Neuromuscular disease

This study tests a new oral polio vaccine (nOPV3) in healthy young children, infants, and newborns in Panama. The goal is to see if it is safe and triggers a strong immune response compared to the current vaccine. About 1,500 participants will receive either the new or existing v…

This study tests two types of inactivated polio vaccine (Sabin strain and wild strain) given in different orders to 480 infants aged 2 months. The goal is to see which sequence triggers the best immune response and causes the fewest side effects. Participants will receive three d…

This pilot study tests whether terazosin, a drug used for high blood pressure, can increase energy levels in people with ALS. Twenty participants will take either terazosin or a placebo for 12 weeks. The goal is to see if the drug is safe and if it raises ATP, a key energy molecu…

This study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the dru…

This Phase 3 trial tests whether DYNE-101 can improve muscle function and daily life in 150 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo by IV every few weeks for 48 weeks. The study measures how quickly people can stand from a ch…

This trial is testing whether a lower weekly dose of the corticosteroid prednisolone (5 mg/kg per week) can be as effective as the standard higher dose (10 mg/kg per week) in preserving motor function in young boys with Duchenne muscular dystrophy (DMD). The study will enroll boy…

This early-phase trial is testing a new treatment called KITE-363 for people with certain autoimmune nerve diseases that have not responded to standard therapies. KITE-363 is a type of CAR T-cell therapy that uses a patient's own immune cells, modified to target and attack faulty…

This early-stage trial tests whether injecting a person's own Schwann cells (taken from a leg nerve) into a damaged major nerve is safe. The study enrolls 30 adults with severe sciatic, brachial plexus, or other major nerve injuries. The goal is to see if the cells can be used wi…

This Phase 3 trial tests a new drug called nucresiran in 125 people with a rare inherited condition that damages nerves (hATTR-PN). The drug is given as a shot under the skin every 6 months and aims to slow nerve damage and improve quality of life. Researchers will compare it to …

This study tests an oral drug called SAT-3247 in 51 boys aged 7 to 10 with Duchenne muscular dystrophy who can still walk. The goal is to find the best dose, check safety, and see if it helps muscle strength. Participants take the drug or a placebo for 12 weeks, and all continue …

This study tests a one-time gene therapy called SGT-003 in 80 boys with Duchenne muscular dystrophy who can still walk. The goal is to see if it helps them move better, like standing up faster and climbing stairs. Participants will receive either the therapy or a placebo first, t…

This trial investigates different types of steroid injections for back, neck, and related pain to see which are most effective and safe. It compares various steroids, doses, and injection sites, including epidural and facet joint injections. The study measures pain relief and fun…

This study tests an investigational drug called ENTR-601-44 in 24 boys and young men with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping a specific part of the gene (exon 44). The trial has two parts: first, to find the safest and most effecti…

This study tests an investigational drug called ENTR-601-45 in people with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 45 skipping). The goal is to check the drug's safety, find the right dose, and see if it can help muscles work better. About 24 ma…

This study tests a drug called tivozanib in people with bile duct cancer that cannot be removed by surgery and has already been treated with chemotherapy. The goal is to find the safest dose and see if the drug can shrink tumors or slow the cancer. Participants take tivozanib by …

This early-stage trial tests an experimental drug called empasiprubart in 60 adults with multifocal motor neuropathy (MMN), a rare condition that causes muscle weakness. The drug is given as an IV infusion, and researchers will use ultrasound to measure nerve thickness, along wit…

This study tests whether a 12-week program of supervised foot and ankle exercises can help people with diabetes who have nerve damage in their feet. The exercises are done in groups at primary care clinics. Researchers will measure changes in symptoms, foot movement, and quality …

This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease and improve muscle function. Participants must be at least 2 years …

This study tests whether adding risdiplam, a daily oral medicine, can help children under 2 with spinal muscular atrophy (SMA) who stopped getting better or started declining after receiving gene therapy. The study will enroll 28 children and measure changes in motor skills over …

This study tests a drug called salanersen in babies with spinal muscular atrophy (SMA) who have already received gene therapy. The drug aims to help the body make more SMN protein, which is needed for muscle function. About 42 babies with two copies of the SMN2 gene will be rando…

This study tests a drug called salanersen in babies who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The drug helps the body make more of a protein that is missing in SMA, which is needed for muscles to work. Researchers want to see if starting t…

This Phase 2 study tests a drug called apitegromab in 60 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle weakness. Participants will receive either the drug or a placebo every 4 weeks for a year. The main goal is to see if the dru…

This study tests a new drug called ARO-DM1 in 78 adults aged 18-65 with type 1 myotonic dystrophy, a genetic muscle disorder. The goal is to check safety and how the body processes the drug. Participants receive either ARO-DM1 or a placebo by IV or injection. It is an early-stage…

This phase 3 trial is testing whether the drug pridopidine can slow the progression of amyotrophic lateral sclerosis (ALS) in 500 adults. Participants will take either pridopidine or a placebo daily for 48 weeks, followed by an open-label extension where everyone receives the dru…

This study tests an experimental drug called ARO-DUX4 in people with facioscapulohumeral muscular dystrophy type 1 (FSHD1), a genetic condition that causes muscle weakness. The main goal is to check the drug's safety and how the body processes it. About 60 adults and teens will r…

This study tests a new biologic drug called ARGX-119 in children aged 5 to 17 with spinal muscular atrophy (SMA). About 60 participants will receive either ARGX-119 or a placebo by IV infusion for 24 weeks, alongside their usual SMA therapy. The goal is to find the best dose and …

This early study tests whether repeatedly opening the blood-brain barrier with ultrasound is safe and might slow ALS. Twenty-three adults with ALS will receive 9 ultrasound sessions over 24 weeks. The first part checks safety; the second looks for signs of slowing the disease.

This study tests an experimental oral drug called ROC-101 in 40 adults with pulmonary arterial hypertension (PAH) or pulmonary hypertension linked to interstitial lung disease (ILD-PH). Participants take the drug alongside their standard care for 24 weeks, with a long-term follow…

This trial is testing an investigational drug called ARV-6723, given as a pill, in people with advanced solid tumors. The drug is designed to help the immune system recognize and attack cancer cells. The study will test ARV-6723 alone and in combination with pembrolizumab, an imm…

This Phase 2 trial is testing an investigational drug called COYA 302 in 120 adults with ALS. The drug combines two immune-modulating agents to reduce inflammation that may damage nerves. Participants will receive either COYA 302 or a placebo for 24 weeks, with an option to conti…

This study tests two different doses of an intravenous immune globulin (IGIV 10%) as maintenance therapy for adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder causing weakness and numbness. About 161 participants will receive either a higher o…

This study tests a single injection of TRCN-1023 into the spinal fluid of adults with ALS. The goal is to see if it is safe and shows early signs of slowing the disease over 24 weeks. Participants are randomly assigned to receive the drug or a placebo, and neither they nor their …

This phase 3 trial tests two different doses of Panzyga, an intravenous immunoglobulin therapy, in children aged 2 to 17 with CIDP, a rare nerve condition causing weakness and disability. The study aims to see which dose better controls symptoms and prevents relapses, using a dis…

This trial tests whether a robot-assisted surgical procedure can relieve chronic pain caused by trapped nerves in the pelvic area. The surgery aims to free the pudendal and inferior cluneal nerves, which may be compressed and causing pain. Researchers will measure changes in pain…

This study tests whether people with ALS who drink an enteral nutrition formula for two weeks before getting a feeding tube (G-tube) will have fewer gastrointestinal issues when tube feeding begins. Participants will drink the formula by mouth first, then use the same formula thr…

This study is testing whether the drug risdiplam can improve motor skills in children under 2 with spinal muscular atrophy (SMA) who have already received gene therapy. The 28 participants will take risdiplam by mouth, and researchers will measure changes in their gross motor ski…

This trial tests whether a new type of deep brain stimulation (DBS) can better relieve chronic pain that hasn't responded to other treatments. Unlike traditional DBS, which delivers constant stimulation, this approach uses a device that adapts stimulation in real time based on br…

This study tests the drug acoramidis in people with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart, weakening it. Over 36 months, researchers measure whether acoramidis improves heart function and structure using cardiac …

This study tests a sensory neuroprosthesis—implanted electrodes that send small electrical currents to remaining nerves—to restore sensation of a missing lower limb in people with diabetic peripheral neuropathy and amputation. The 15 participants will also have the option of musc…

This phase 3 trial tests whether givinostat, an oral drug, can slow muscle decline in boys aged 9 to 18 with Duchenne muscular dystrophy who can no longer walk. Participants receive either givinostat or a placebo for 18 months. The main goal is to see if the drug helps maintain u…

About 10-20% of people with ALS have a specific antibody (anti-NRIP) in their blood that is linked to faster disease progression. This trial tests whether removing that antibody through plasmapheresis — a procedure that filters the blood — can slow the decline. Twenty participant…

This trial tests whether placing a piece of amniotic membrane soaked in a stem-cell-derived solution (secretome) on the nerve during carpal tunnel release surgery can improve symptom relief and nerve function. Adults with moderate-to-severe carpal tunnel syndrome that hasn't impr…

This early-stage trial is testing an experimental oral drug, HPB-092, in people whose acute myeloid leukemia (AML) has returned or not responded to standard treatments. The drug is designed to block two proteins, FLT3 and IRAK4, that help leukemia cells grow and survive. The stud…

This study investigates whether adding melatonin to standard diabetes care can improve symptoms of diabetic peripheral neuropathy, a common complication causing pain and numbness in the feet and hands. The trial includes 60 adults aged 40-75 with type 2 diabetes and diagnosed ner…

This study follows 250 boys (ages 2 and older) with Duchenne muscular dystrophy who are taking vamorolone (AGAMREE). Researchers will monitor side effects, growth, bone health, heart function, and quality of life over time. The goal is to better understand the long-term safety an…

This study looks at the long-term safety of an experimental drug called LY4256984 for people with ALS. It enrolls up to 32 participants who have completed a prior ALS trial. The study will follow them for at least 96 weeks to see how well the drug is tolerated over time.

This study tests the long-term safety of a drug called LY4256984, given via spinal injection, in people with ALS. It includes 32 participants who completed an earlier study and will be followed for about 96 weeks. The main goal is to see how many people have serious side effects …

This study tests a medicine called surlorian in 28 adults with a rare genetic muscle disease (RYR1-related myopathy) that causes weakness. The goal is to see if surlorian improves muscle strength and function, like standing from a chair or walking. Participants will be randomly a…

This early-phase trial tests a single injection of gene therapy (ELP-02) into the spinal fluid of 8 children and young adults with CMT4J, a rare genetic nerve disease that causes muscle weakness. The therapy delivers a working copy of the FIG4 gene to nerve cells, potentially slo…

This study tests whether a weekly under-the-skin antibody treatment (XEMBIFY) provides similar blood levels as a standard IV infusion (Gamunex-C) given every 3 weeks in people with CIDP, a chronic nerve disorder. About 40 adults who are stable on IVIG will first receive Gamunex-C…

This study tests a brain implant that records and stimulates brain areas to help adults with severe paralysis (from spinal cord injury, ALS, or stroke) control assistive devices like computers or wheelchairs. The main goal is to check safety, and researchers will also see if part…

This trial tests a single injection of MELPIDA, a gene therapy that delivers a working copy of the AP4M1 gene directly into the spinal fluid of children with SPG50. SPG50 is a rare genetic condition that causes muscle stiffness, intellectual disability, and developmental delays. …

This study tests two experimental drugs, sapanisertib and serabelisib, combined with standard therapy for people with HR+/HER2- advanced or metastatic breast cancer. The goal is to find safe doses and see if the combination can shrink tumors or slow the disease. About 32 adults w…

This study tests whether a special probiotic supplement can change fat-related molecules in the blood of people with ALS and frontotemporal dementia (FTD). Researchers will compare those taking the probiotic to those taking a placebo over 24 weeks. The goal is to see if the probi…

This study tests a single intravenous dose of a gene therapy called GNR-097 in boys aged 4 to 9 with Duchenne muscular dystrophy (DMD). The therapy uses a harmless virus to deliver a shortened version of the dystrophin gene, which is missing or faulty in DMD. The trial aims to se…

This study is testing whether stem cells taken from a person's own bone marrow and then given back through the veins and nose can improve brain and nerve function. It includes 500 people with various conditions like stroke, Parkinson's, Alzheimer's, and nerve damage. The goal is …

This early-stage study tests a new gene-editing medicine called PBGENE-DMD in 18 boys aged 2 to 7 with Duchenne muscular dystrophy. The treatment aims to correct the genetic mistake that causes the disease, potentially restoring muscle strength. Researchers are first checking if …

This trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare and life-threatening genetic disorder that damages the nervous system. The treatment uses a lentivirus to deliver a working copy of the ARSA gene directly into the spinal fluid and bloodstream. Up to 1…

This study tests an investigational drug called LTX-002 in people with amyotrophic lateral sclerosis (ALS). The drug is designed to target a specific genetic message linked to the disease. The main goal is to check the drug's safety and tolerability, and to see how it moves throu…

This phase 2 trial tests a combination of three drugs—carfilzomib, cyclophosphamide, and dexamethasone—in 20 people newly diagnosed with POEMS syndrome, a rare disorder affecting nerves and blood. The goal is to see if the regimen can reduce abnormal proteins and improve symptoms…

This study tests whether a processed nerve graft from a donor (Avance Nerve Graft) works as well as using a piece of the patient's own leg nerve to repair damaged nerves in the arm. About 178 adults with upper arm nerve injuries will be randomly assigned to receive either the don…

This study tests whether zanubrutinib, a drug used for certain blood cancers, can improve nerve function in people with anti-MAG neuropathy—a rare condition causing numbness, weakness, and balance problems. About 50 adults in Italy will take the drug for 12 months. Researchers wi…

This study tests a brain-computer implant (Stentrode) for people with paralysis from motor neuron disease or similar conditions. The device is placed in a blood vessel near the brain and aims to let users control computers or assistive devices with thought. The trial focuses on s…

This study tests whether a 4-month structured telerehabilitation program, delivered via video calls and an app, can reduce deaths and hospital readmissions in heart patients who have Post-Intensive Care Syndrome (PICS). About 326 adults aged 30-75 who were in the ICU for at least…

This Phase 2 trial compares the drug dolutegravir alone versus dolutegravir combined with tenofovir in 146 adults with HTLV-1 infection and related symptoms. The main goal is to see if the combination lowers the virus level more effectively and improves pain, spasticity, and musc…

This study tests a new medicine called DYNE-251 in boys aged 4 to 18 with Duchenne muscular dystrophy (DMD) who can still walk. The goal is to see if it helps them move better and slows muscle damage. Participants will receive either the drug or a placebo every 4 weeks for about …

This study is testing whether a drug called tadalafil (often used for erectile dysfunction) combined with a home cycling program can help boys with Duchenne muscular dystrophy. The drug aims to improve blood flow to muscles, which is often poor in DMD, while exercise builds stren…

This early study tests a brain-computer implant placed through blood vessels to help people with ALS control external devices using their thoughts. Ten participants with ALS and arm weakness will be followed for safety and device function. The goal is to restore some independence…

This early-phase trial is testing a new cell therapy called QH103 for people with severe autoimmune diseases like multiple sclerosis and myasthenia gravis that haven't responded to standard treatments. The therapy uses specially engineered immune cells to target and attack the fa…

This study tests an experimental drug called TP04HN106 in 60 people with ALS. Half receive the drug, half receive a placebo (saline), and all take riluzole tablets as standard care. The goal is to see if the drug is safe and can slow the decline in physical function measured by t…

This study tests whether giving ALS patients 20% more calories than their body needs (via a feeding tube) can slow the disease. Researchers will measure a nerve damage marker in the blood over 6 months. 76 adults with ALS who already use a feeding tube will be randomly assigned t…

This study tests whether an ultra-high-caloric diet made almost entirely of fat can help people with ALS live longer and slow the disease. Researchers will compare the diet to a low-fat placebo in 392 participants. The main goal is to see if the diet extends survival without the …

This study compares two treatments for CIDP, a nerve condition that causes weakness and numbness. It involves 20 adults who did not get better with steroids. One group receives a blood-filtering procedure called immunoadsorption, and the other receives standard antibody infusions…

This study tests whether GLP-1 receptor agonists, a class of diabetes medications including semaglutide, can improve heart nerve damage in people with type 2 diabetes. 60 adults will receive either standard care or standard care plus a weekly injection for 6 months. Researchers w…

This early-stage trial tests a gene therapy called scAAV9/JeT-GAN for giant axonal neuropathy (GAN), a rare, fatal nerve disease in children. The therapy is injected directly into the left vagus nerve to target autonomic nervous system symptoms. Only 4 people who have already rec…

This early-stage trial tests a new drug called TOS-358 in women with advanced HR+ HER2- breast cancer that has a specific mutation in the PIK3CA gene. The study aims to find the safest dose and see if the drug works alone or with standard treatments like fulvestrant and CDK4/6 in…

This phase 3 study tests a drug called salanersen (BIIB115) in people aged 15-60 with spinal muscular atrophy (SMA). The drug is designed to help the body produce more of a protein needed for nerve and muscle function. Participants receive an injection into the spine once a year …

This study tests a small absorbable cap placed over cut nerve endings during finger amputation surgery. The goal is to prevent painful neuromas (nerve balling) that often cause chronic pain. Twenty adults who have had a traumatic finger amputation will be followed for up to a yea…

This study tests whether a special exercise program that combines rhythm and multitasking (called RYMA) can reduce falls, fractures, and heart problems in people aged 65 and older with type 1 or type 2 diabetes. Participants will be screened for fall risk, bone health, nerve func…

This study tests a brain-computer implant (BCI) that lets people with ALS or motor neuron disease control digital devices using only their thoughts. Ten participants will receive the implant, and researchers will monitor safety and how well the device works over one year. The goa…

This phase 2 trial tests whether anakinra (Kineret), an anti-inflammatory drug, can improve hearing in people with Meniere's disease or autoimmune inner ear disease who cannot take steroids. About 57 participants will receive daily injections of anakinra or a placebo for 42 days,…

This study tests whether a 3-week intensive summer camp combining motor skill training and strength exercises can improve movement in children with spinal muscular atrophy (SMA). Twenty children aged 5-17 who are already on stable disease-modifying therapy will attend 6-hour sess…

This study tests a new type of shoe, called the LEEF-shoe, designed to prevent foot ulcers in people with diabetes and nerve damage. About 30 adults will wear both the LEEF-shoe and standard orthopedic shoes for several weeks to compare how well they relieve pressure on the feet.…

This study tests whether giving botulinum toxin (Botox) injections early can prevent shoulder bone deformities in babies with obstetrical brachial plexus palsy, a nerve injury at birth. About 62 babies aged 10-11 months will receive either the injection or a sham procedure. The g…

This study tests a new brain-computer interface called MindEx that uses four small chips implanted in the brain to help people with severe paralysis control a computer or tablet just by thinking. The goal is to improve independence and quality of life by allowing users to type, p…

This study tests a program called PICU Up! that helps critically ill children start moving earlier while in the pediatric intensive care unit. The program includes daily activity plans, better sedation management, and family involvement. Researchers want to see if this approach r…

This study tests a temporary electrical stimulation system that delivers a single, one-hour therapy to help heal acutely injured peripheral nerves. Researchers will enroll 110 people with recent nerve injuries to see if the treatment is safe and improves sensory recovery. The app…

This study tests whether using technology like handgrip games, virtual reality, and in-bed cycling, along with family help, can prevent muscle weakness in ICU patients on breathing machines. 138 critically ill adults will be split into three groups: one gets the tech-assisted pro…

This study follows 15 adults (ages 22–90) with severe, long-term balance disorders caused by inner ear damage. Participants have already received a vestibular implant, a device that electrically stimulates the balance nerve to help restore steadiness and clear vision during movem…

This study tests several already-approved drugs to see if they can slow down motor neuron disease (MND) and help people live longer. About 1150 adults with MND will be randomly assigned to receive one of the study drugs or a placebo. The trial uses a flexible design, allowing ine…

This early-phase trial tests whether injecting lab-grown muscle cells (MyoPAXon) into the foot is safe for adults with Duchenne muscular dystrophy who can no longer walk. Eight participants will receive the cells along with an immunosuppressant drug to prevent rejection. The main…

This study looks at whether drinking non-alcoholic red wine, which is rich in natural compounds called polyphenols, can help older adults keep their thinking sharp and muscles strong. Researchers will give 72 people aged 60-74 either non-alcoholic red wine or water, both as part …

This study tests whether a substance called polyethylene glycol (PEG) can help reconnect severed nerves in the arm more effectively than standard surgery. About 40 adults with a recent, complete nerve injury from trauma will receive either PEG plus standard repair or standard rep…

This early study is testing a drug called triheptanoin (Dojolvi) in children with pyruvate dehydrogenase complex (PDC) deficiency, a rare genetic condition that affects energy production. The trial will enroll 6 children aged 1 to 18 and look at safety, side effects, and changes …

This study tests whether taking N-acetylcysteine (NAC) and EH-301 together with the standard drug riluzole can slow down amyotrophic lateral sclerosis (ALS). About 90 people with early-stage ALS will receive either the active combination or a placebo for 6 months. After that, eve…

This early-stage study tests a single dose of a gene therapy called CTx1000 in 15 people with ALS. The main goal is to see if it is safe and tolerable. Participants must have had ALS for 2 years or less and be on stable standard medications. This is not a cure; it aims to control…

This study looks at whether taking a small piece of ligament during routine carpal tunnel surgery can help diagnose transthyretin amyloidosis, a serious condition that often goes unnoticed until it affects the heart. Researchers will compare this new biopsy method to the standard…

This study aims to translate an English questionnaire used to diagnose carpal tunnel syndrome into Thai and check if it works well. Researchers will enroll 80 adults at Siriraj Hospital who have been diagnosed with carpal tunnel syndrome. Participants will answer the new Thai que…

This study is testing whether a new approach—using ultrasound to guide a tiny needle to collect fluid near the ulnar nerve, then analyzing it for leprosy bacteria DNA—can more accurately diagnose leprosy and confirm when it's cured. Researchers will follow 108 participants for on…

This study is testing a handheld device called mScan that uses a tiny, painless electrical current to measure muscle health. Researchers want to see if it can give similar results to an MRI, but faster and more conveniently. The study involves 150 adults with and without muscle d…

This study tests whether a wearable device called SleepImage can accurately detect respiratory failure in people with ALS (Lou Gehrig's disease). Researchers will compare the device's readings with standard overnight sleep tests in 15 patients. The goal is to find a simpler way t…

This study tests a portable DNA device called MinION that can identify bacteria and fungi in foot wounds of people with diabetes in under 4 hours. Standard lab cultures take longer and may miss some germs. The goal is to see if this faster method gives a more complete picture of …

This phase 3 trial tests whether acoramidis can prevent or delay transthyretin amyloidosis (ATTR) in 587 adults who carry a faulty gene but have no symptoms yet. ATTR causes sticky plaques to build up in the heart and nerves, leading to heart failure and nerve damage. Acoramidis …

This study tests if special sensor-equipped insoles can help prevent foot ulcers from coming back in people with diabetes and nerve damage. About 400 high-risk participants will use the insoles alongside standard care or just standard care alone. The goal is to see if the insoles…

This phase 3 trial tests whether adding daily calcitonin injections to standard shingles treatment can prevent postherpetic neuralgia, a painful nerve condition that can last for months or years. Researchers will enroll 168 adults aged 50 and older who have had a shingles rash fo…

About 30% of people who have surgery for a herniated disc still suffer from nerve pain afterward. This study tests whether applying a gel made from the patient's own blood (platelet-rich fibrin) around the nerve during surgery can prevent that chronic pain. The trial will enroll …

This study tests whether taking fish oil supplements can prevent nerve damage in the hands and feet caused by oxaliplatin chemotherapy in colorectal cancer patients. 120 participants will receive either fish oil or corn oil (placebo) during their treatment. The goal is to see if …

This study tests whether cooling and compressing the arms and legs during taxane chemotherapy can prevent peripheral neuropathy—a common side effect causing numbness, tingling, or pain. About 777 cancer patients will be randomly assigned to one of three approaches: cryocompressio…

This study tests whether taking mecobalamin (a form of vitamin B12) can prevent or reduce nerve damage caused by taxane chemotherapy. About 326 adults with various solid tumors will take mecobalamin tablets or receive no preventive treatment during their chemo. The goal is to see…

This study tests whether wearing tight-fitting surgical gloves and compression knee socks during chemotherapy can prevent nerve damage (neuropathy) in breast cancer patients. The idea is that compression narrows blood vessels, reducing the amount of chemo drugs reaching the hands…

This study tests whether soaking feet in cold salt water can prevent nerve damage caused by the chemotherapy drug paclitaxel in breast cancer patients. Thirty women will be split into three groups: cold salt water foot baths, cold unsalted water foot baths, or standard care. The …

This phase 2 trial tests whether drinking tart cherry juice can prevent nerve damage (neuropathy) caused by the chemotherapy drug paclitaxel in breast and ovarian cancer patients. About 86 participants will be randomly assigned to receive either a high or low dose of the juice du…

This pilot study tests whether VM100, a nutritional supplement made from ten natural ingredients, can improve quality of life for people with Duchenne muscular dystrophy (DMD). About 20 participants aged 8 and older will take VM100 or a placebo for 10 weeks. Researchers will meas…

This study tests a five-week tongue exercise program for 20 people with ALS to see if it can help with swallowing and speech. Participants press their tongue against a device at home five days a week, with weekly check-ins via telehealth. Researchers will measure tongue strength …

This trial tests whether an experimental tablet called HL-1186 can reduce the persistent nerve pain that sometimes lingers after a shingles rash heals, a condition known as postherpetic neuralgia. About 200 adults with this pain will receive either HL-1186, a placebo, or the stan…

This study tests whether psilocybin (the active ingredient in magic mushrooms) can help ease depression and improve quality of life in people with ALS. Researchers will enroll 24 adults with ALS and depressed mood to see if the therapy is feasible and reduces symptoms. The goal i…

This study tests whether cycling while using a virtual park app can slow the loss of motor function in children and teens with neuromuscular diseases like muscular dystrophy. Twenty-two participants will first receive standard care for 3 months, then use the virtual park bike 3 t…

This trial tests whether adding kinesio tape to somatosensory motor control exercises improves pain, lower-back flexibility, balance, and daily function in people with sciatica. Adults aged 35-50 with a herniated disc in the lower back and leg pain lasting 3-6 months will receive…

This small study at MD Anderson tests whether peripheral nerve stimulation (PNS) can reduce pain from chemotherapy-induced peripheral neuropathy (CIPN). Ten adults with chronic leg pain from past chemo will receive PNS, where thin wires are placed under the skin near nerves to de…

This study tests whether psilocybin (the active ingredient in magic mushrooms) is safe and tolerable for veterans with spinal cord injury who also have depression. Thirty participants will receive low, medium, or high doses and be monitored for side effects like pain, muscle spas…

This trial tests whether dry needling and low-level laser microcurrent electrical stimulation can reduce pain and inflammation in people with acute myofascial pain syndrome in the upper trapezius muscle. Participants are randomly assigned to receive one of these treatments, a com…

This study tests whether a single injection of RE104 can reduce symptoms of depression or mixed depression and anxiety in people with adjustment disorder caused by a serious medical illness like cancer, ALS, multiple sclerosis, Parkinson's disease, or idiopathic pulmonary fibrosi…

This study tests whether scrambler therapy, a device that sends electrical signals to the skin, can reduce pain, numbness, and tingling from chemotherapy-induced peripheral neuropathy. About 90 adults with persistent nerve pain will receive either real or sham treatment for 10 se…

This trial tests whether adding focal tendon vibration to a two-week intensive rehabilitation program can improve balance in adults with Charcot-Marie-Tooth disease type 1A (CMT1A). CMT1A causes progressive muscle weakness and poor balance, often leading to falls. Participants wi…

This trial tests whether taking Ceylon cinnamon supplements for six months can reduce pain and improve blood sugar, cholesterol, and body weight in adults with painful diabetic peripheral neuropathy. Participants are randomly assigned to receive either cinnamon capsules or a plac…

This study tests a new drug called (2R,6R)-hydroxynorketamine (HNK) for chronic nerve pain in the arms or legs. 25 adults will receive a single infusion of HNK, ketamine, or a placebo in random order, with 5 weeks between each. Researchers will measure pain levels, sleep, and dai…

This trial tests a wearable-sensor system called MINDGAPS that tracks eye and body movements during balance exercises. Researchers want to see if it can improve dizziness and stability in older adults with vestibular hypofunction — a common cause of falls. The study also checks w…

This trial tests whether a gentle electrical current applied to the brain, called transcranial direct current stimulation (tDCS), can boost language skills in adolescents and young adults with Down syndrome when paired with speech therapy. Thirty-six participants aged 12 to 21 wi…

This study tests whether a spinal cord stimulator can reduce pain and improve nerve function in people with diabetic peripheral neuropathy. Sixty adults with pain that hasn't responded to standard treatments will be randomly assigned to receive the implant or standard care. Resea…

This trial tests whether three types of nerve-gliding exercises — called neurodynamics — can improve hamstring strength and endurance in athletes aged 18 to 30 who play organized sports or train regularly. The exercises aim to improve nerve mobility and reduce pain, and the study…

This study tests whether benfotiamine, a form of vitamin B1, works better than a combination of vitamins B1, B6, and B12 at reducing numbness, pain, and other sensory nerve symptoms in people with diabetic peripheral neuropathy. Participants take either benfotiamine or the vitami…

This study is testing if a non-invasive brain stimulation method called HD-tDCS can help people with ALS. The goal is to see if it can improve muscle control and breathing by calming overactive brain cells. The study will involve 80 adults with ALS and will measure changes in bra…

This phase 3 trial tests whether an extended-release (once-daily) form of pregabalin works as well as the standard immediate-release (twice-daily) form for relieving nerve pain caused by diabetic peripheral neuropathy or postherpetic neuralgia. About 348 adults aged 18 to 75 with…

This trial tests a 12-session telecare program for children aged 7 to 17 with limb-girdle muscular dystrophy. The group-based intervention focuses on social cognition, emotional management, and coping strategies to improve quality of life and reduce symptoms. Participants attend …

This study investigates whether low-frequency pulsed electromagnetic field (PEMF) therapy can improve vibration perception and protective sensation in people with diabetic foot ulcers. Forty-two participants with type 2 diabetes and Wagner grade 1 ulcers will receive either real …

This study compares three methods to reduce pain during corticosteroid injections for arthritis, tendinopathy, or nerve compression in the hand, wrist, or elbow: no anesthesia, a vibrating device, or a cooling spray. Each participant will receive two of the three methods to see w…

This study tests whether a supplement called Normast3 can reduce nerve pain and improve nerve function in adults with diabetic polyneuropathy, a common complication of diabetes. About 40 participants will take either Normast3 or a placebo twice daily for 12 weeks, while researche…

This study investigates whether matrix rhythm therapy, a device that delivers gentle vibrations to the hand and arm, can reduce symptom severity and improve hand function in people with mild-to-moderate carpal tunnel syndrome. Participants receive either active or sham therapy tw…

This pilot study tests whether the Voiceitt app can help people with speech impairments caused by conditions like cerebral palsy, stroke, ALS, or Parkinson's disease communicate more effectively. Participants use the app to see if it improves speech clarity and daily communicatio…

This study tests a new treatment called ACTIVE that combines counseling (Acceptance and Commitment Therapy) with balance exercises for people with mild traumatic brain injury. Researchers want to see if it helps reduce dizziness and anxiety better than standard balance therapy al…

This study tests whether people can use a small, portable device at home to stimulate the vagus nerve through the ear. It aims to see if this approach is practical and acceptable for managing nerve pain caused by chemotherapy. The study includes 36 adults who have had platinum or…

This study tests whether a non-invasive brain stimulation technique called rTMS can reduce nerve pain, depression, and sleep problems in people with spinal cord injury. Researchers will compare two different rTMS protocols against a placebo in 63 adults. Participants continue the…

This study compares standard carpal tunnel release surgery to an extended version that also releases a ligament in the forearm. Many people still have symptoms after standard surgery, possibly because the nerve is also compressed higher up. The trial will enroll 110 adults with c…

This study looks at whether a high-dose capsaicin patch (made from chili pepper extract) can relieve long-lasting tailbone pain that hasn't responded to other treatments. Researchers will review medical records from 106 patients treated over six years to see how well the patch wo…

This study tests a device called Walkasins, worn like an ankle strap and insole, that gives gentle vibrations to help people with peripheral neuropathy feel their feet better. The goal is to see if it improves walking, balance, and daily physical activity. Eighteen adults aged 60…

This study tests whether describing a common pain shot (epidural steroid injection) in a very positive way helps reduce leg pain more than a neutral description. Researchers will compare three groups: positive framing of the shot, neutral framing of the shot, and neutral framing …

This trial tests a wearable robot called ENMS-BSF that combines soft muscles, electrical stimulation, and balance feedback to correct foot drop and foot inversion after a stroke. Sixty people who had a stroke at least six months ago and have mild-to-moderate walking problems will…

This study tests whether a combination of two existing drugs, nortriptyline and topiramate, can reduce vertigo and other symptoms in people with Meniere's disease. Forty adults with active Meniere's will take the drugs or a placebo for 8 weeks. The goal is to see if this approach…

This study tests whether a single ultrasound-guided injection of medicine around compressed leg nerves can relieve numbness, pain, and weakness in people with diabetes. 266 participants will be randomly assigned to get the real injection or a sham (fake) injection, and neither th…

This study tests whether nerve gliding exercises, tendon gliding exercises, or a combination of both can reduce pain and improve hand function in people with mild to moderate carpal tunnel syndrome. Participants will be randomly assigned to one of three groups and followed for ch…

This study tests whether a personalized exercise program can improve balance and physical function in adults with rare neuromuscular disorders like Charcot-Marie-Tooth disease, facioscapulohumeral muscular dystrophy, and myotonic dystrophy type 1. Participants will receive a 12-d…

This study tests whether shortwave therapy can relieve symptoms of diabetic peripheral neuropathy, a common complication causing pain and numbness. About 202 adults with diabetes will receive either real shortwave treatment or a sham device for 20 minutes daily over five days. Re…

This study looks at whether electroacupuncture (a type of acupuncture with mild electric pulses) can help relieve pain from shingles and reduce the chance of long-term nerve pain. About 228 adults with shingles will be split into three groups: one gets electroacupuncture plus sta…

This study tests whether a six-week at-home light therapy program is practical and helpful for childhood cancer survivors who have nerve pain or numbness from chemotherapy. Twenty survivors aged 5 and older will use a light therapy device on their symptoms for 15 minutes, three d…

This early study tests a virtual reality rehabilitation device for children with spinal muscular atrophy (SMA), a genetic condition that causes progressive muscle weakness. The device uses games, muscle sensors, and a grip-strength ball to make home exercises more engaging. Resea…

This early-stage study tests whether an injection of RP-008 followed by a daily varenicline pill is safe and can reduce pain for people with trigeminal neuralgia, a condition causing severe facial pain attacks. About 24 adults aged 18-80 with classic or idiopathic trigeminal neur…

This study tests a new type of breathing machine (volume-targeted ventilation) for people with slowly worsening muscle diseases who already use a standard breathing machine at night. The goal is to see if it can better control carbon dioxide levels and improve sleep comfort. Abou…

This study compares two types of nerve-stretching exercises (tensioner and slider) for people with sciatica. Eighty participants with leg pain lasting 3 to 12 months will receive one of the techniques plus standard physical therapy. Researchers will measure pain, disability, and …

This study tests whether a 10-week virtual exercise program can reduce nerve pain and improve daily activities in cancer survivors who have persistent nerve damage from chemotherapy. About 240 participants will either receive the program or usual care. The program includes person…

This study compares a targeted nerve block injection to usual pain treatment for people with sudden sciatica in the emergency department. About 100 adults will be randomly assigned to receive either the nerve block or standard care. Researchers will measure pain levels and walkin…

This study tests whether standard carpal tunnel surgery—cutting a ligament in the wrist—actually relieves symptoms like numbness and pain better than a pretend surgery where the ligament is not cut. About 180 adults with carpal tunnel syndrome will be randomly assigned to real or…

This small study tests whether a gentle, non-invasive brain stimulation technique called tACS can reduce nerve pain in people with spinal cord injury. Fourteen participants will receive either real or sham stimulation for 20 minutes. Researchers will measure pain levels and heart…

This small pilot study tests whether using a hand-held vibration device for just 3 minutes each day can safely reduce nerve pain and numbness in the hands caused by chemotherapy. Sixteen adults who finished chemotherapy at least 60 days ago will try the therapy at home. The main …

This study tests a home-based exercise program for people who were hospitalized with severe COVID-19. Participants use a smartphone app to guide them through high-intensity interval training and other exercises. The goal is to see if this approach improves walking ability, fitnes…

This study will give a steroid and numbing injection to 60 people with carpal tunnel syndrome, half of whom also have type 2 diabetes. Researchers will track pain, nerve function, and hand strength for 12 weeks to see if the diabetes group recovers as well as those without diabet…

This study tests whether balance rehabilitation that changes how patients see (closing eyes, using a mask, or projecting moving dots) can improve walking stability better than standard rehab with eyes open. It includes 40 adults with chronic nerve damage who have trouble with bal…

This study tests whether a common back operation (microdiscectomy) actually relieves sciatica leg pain better than a pretend surgery. 122 adults with a herniated disc and persistent leg pain will be randomly assigned to get either the real operation or a sham procedure. Neither t…

This study tests two stretching techniques—post-isometric relaxation and active isolated stretching—in 40 adults with piriformis syndrome, a condition that causes buttock and hip pain. Over two weeks, participants will receive six sessions of one stretch type plus standard physio…

This study tests whether a gel containing pregabalin, applied through the skin using ultrasound (phonophoresis), combined with calf muscle stretching, can reduce nerve pain and improve walking in people with grade III knee osteoarthritis. The trial includes 35 adults aged 40-70 w…

This study tests whether playing virtual reality games can help people with Duchenne muscular dystrophy improve their arm and hand function. 36 participants will either receive standard physiotherapy or the same therapy plus VR games using a Meta Quest 3 headset. The games are de…

This study is testing whether combining two types of electrical stimulation can help people who have had a stroke lift their toes while walking. Participants will receive mild electrical currents on their scalp and leg while doing walking exercises. The goal is to see if this com…

This study is testing whether different types of NSAIDs (like ibuprofen, diclofenac, and meloxicam) work better for specific pain types in people with frozen shoulder. 120 adults with shoulder pain and stiffness will take one of these drugs along with home exercises for two weeks…

This study tests whether giving calcitonin early after a spinal cord injury can lower the chance or severity of nerve pain. About 126 adults with a recent spinal injury will receive either calcitonin or a placebo. Researchers will track pain levels and side effects for up to a ye…

This study looks at whether adding ultrasound-guided shoulder injections to a home exercise program can reduce pain and improve movement in people with frozen shoulder. About 68 adults with chronic shoulder pain will receive either one type of injection or a combination, and be f…

This pilot study tests a mechanical in-exsufflator (MIE) device that helps ALS patients cough more effectively by pushing air in and then pulling it out. The goal is to see if daily use for four weeks can maintain or improve lung function. The study will enroll 36 adults with ALS…

This study tests a powered leg brace called the Nomad in 36 people who have trouble walking due to conditions like stroke, multiple sclerosis, or spinal cord injury. Participants will use the brace at home for three months to see if it improves their walking speed, balance, and q…

This study tests whether a modified version of the muscle relaxant metaxalone (640 mg) can reduce low back and leg pain in people with acute sciatica or spinal stenosis. Fifty adults aged 18 to 80 with new-onset pain will take either the drug or a placebo for 7 days. Researchers …

This study tests whether a simple head-and-body movement called the Epley maneuver can quickly relieve dizziness from benign paroxysmal positional vertigo (BPPV) in the emergency department. About 118 adults with BPPV will either get the real maneuver or a fake one. Researchers w…

This study compares two numbing drug recipes for a nerve block given before leg surgery. One group gets a mix of lidocaine and bupivacaine, while the other gets bupivacaine alone. Researchers want to see which combination works faster and provides better pain control after the op…

This study tests whether psilocybin (the active ingredient in magic mushrooms) can safely reduce depression in adults with COPD, ALS, MS, or atypical Parkinson disease who have at least 6 months to live. Participants receive either two moderate-to-high doses (15mg then 25mg) or t…

This study tests two types of foot and ankle exercises to see which one better helps balance, walking, and fall risk in people with diabetic peripheral neuropathy (nerve damage in the feet). Researchers will compare exercises for the small muscles in the foot with 3D ankle-foot e…

This study tests whether combining brain stimulation (rTMS) with virtual reality mirror therapy can reduce hard-to-treat nerve pain. 64 adults with chronic neuropathic pain that hasn't responded to drugs will receive either real or sham mirror therapy alongside rTMS. The goal is …

This study compares two exercise approaches for dead butt syndrome, a condition where the gluteal muscles become inactive, causing pain and discomfort. Forty participants will be randomly assigned to either gluteal activation exercises alone or combined with core strengthening. T…

This study tests whether adding a specific leg-rotation technique to standard physical therapy can better relieve sciatica caused by disc problems in the lower back. Researchers will measure pain, flexibility, and daily function in 70 adults aged 18 to 50. The goal is to see if t…

This study looks at whether a custom mouthguard (occlusal splint) can reduce pain and improve muscle function in people with teeth grinding (bruxism) and related jaw and neck muscle pain. About 40 adults will be split into two groups: one gets the mouthguard right away, the other…

This study tests whether a mobile app can teach family caregivers safe massage techniques to help cancer survivors with long-term nerve pain and numbness caused by chemotherapy. About 300 participants (survivors and their caregivers) will use the app at home. The goal is to reduc…

This study tests whether low-dose naltrexone (LDN) can safely reduce pain from diabetic neuropathy, a common complication of diabetes. Thirty-five adults who have not found relief from standard treatments will receive LDN and a placebo in random order. The goal is to see if LDN o…

This study tests whether combining spinal cord stimulation with a rehabilitation program can reduce chronic nerve pain in the legs and improve physical function. Ten adults aged 18-60 with nerve pain lasting over 6 months will receive the treatment bundle. The goal is to see if t…

This study tests a new type of deep brain stimulation called 'Burst-DBS' for people with long-term facial pain or pain after a stroke. Twenty adults will have electrodes placed in the thalamus (a pain-related brain area) and try both standard and burst stimulation patterns. The g…

This study tests whether eye drops made from cord blood plasma can reduce pain in people with long-term nerve-related eye pain. Twenty adults with persistent eye pain and abnormal corneal nerves will receive the drops. Researchers will measure pain levels and nerve health to see …

This study tests three different injections—exosomes, platelet-rich plasma (PRP), and steroids—to see which best eases long-term nerve pain after shingles. 45 adults with pain lasting at least 3 months will get four treatments every two weeks. Researchers will measure pain, quali…

This study tests whether a whole-food, plant-based diet (no added oils) can reduce painful diabetic neuropathy better than standard medication. Forty adults with type 2 diabetes and nerve pain will be randomly assigned to either the diet or medication optimization. The main goal …

This study tests whether injecting a long-lasting numbing medication (Exparel) into the side of the nose can reduce severe face and headache pain. About 15 adults with conditions like migraine or cluster headache will receive either the drug or a placebo, then switch groups. The …

Stanford University is testing a non-invasive device that uses pulsed radiofrequency to treat chronic nerve pain after injury. The device sends magnetic pulses through the skin to target the affected nerve. This Phase 2/3 trial will enroll 95 adults to see if the treatment reduce…

This study tests a noninvasive spinal cord stimulation (tSCS) to improve walking and reduce muscle stiffness in 15 adults with hereditary spastic paraplegia (HSP), a rare nerve condition. Participants receive stimulation through skin electrodes on the lower back twice a week for …

This study tests whether taking amino acid supplements, along with a special diet and exercise program, can help people with facioscapulohumeral muscular dystrophy (FSHD) gain muscle, lose fat, and improve physical abilities like walking and strength. About 48 adults with FSHD an…

This pilot study tests whether Tai Chi, alpha-lipoic acid supplements, and acupuncture can help ease chemotherapy-induced peripheral neuropathy (nerve pain, numbness, tingling) in people with gastrointestinal cancer. About 60 participants will try one or more of these approaches …

This early study tests a small device that sends gentle electrical pulses to nerves to relieve severe, long-lasting knee, elbow, or shoulder pain. About 30 adults with chronic pain will first try a temporary implant for up to 8 hours. If it works well, they can get a permanent ve…

This study tests whether using a long-acting local anesthetic called Exparel, along with education about opioids, can help people avoid taking opioid painkillers after carpal tunnel release surgery. About 64 adults with carpal tunnel syndrome will be split into two groups: one ge…

This study tests a gel called DYNAVISC placed around the nerve during repeat carpal tunnel surgery to see if it reduces pain and tingling better than standard surgery alone. About 50 adults with ongoing hand pain after a prior carpal tunnel release will take part. Results are mea…

This study tests whether adding peripheral magnetic stimulation to balance exercises can lower fall risk in older adults with diabetic polyneuropathy. Forty participants will receive either real or sham magnetic pulses to their feet while doing balance training. The goal is to se…

This phase 3 trial compares a capsaicin patch (applied once) to the daily pill duloxetine for treating painful nerve damage caused by chemotherapy. About 274 people whose nerve pain persists at least a month after chemo will be randomly assigned to one treatment. The goal is to s…

This study tests a powered neck brace (exoskeleton) in 10 people with ALS who have neck weakness. Participants will do simple tasks while wearing the device to see if it improves head movement and is comfortable. The goal is to help patients regain the ability to move their heads…

This study tests a smartphone app called Tuntun Rehabilitation Assistant to help people who have been in the intensive care unit (ICU) regain strength and mobility. About 70 adults from two hospitals in South Korea will use the app for daily 30-minute rehab sessions. Researchers …

This study compares two treatments—kinesiotaping (a special tape) and peloidotherapy (mud therapy)—plus home exercises, against exercises alone for people with mild-to-moderate carpal tunnel syndrome. 90 adults will be randomly assigned to one of three groups. The goal is to see …

This study tests whether pairing non-invasive brain and spinal cord stimulation with hand exercises can improve hand strength and dexterity in veterans with ALS. Thirty-two participants will first undergo a personalized optimization period, then receive two weeks of the combined …

This study tests whether whole-body electrical muscle stimulation (WB-EMS) can help adults with neuromuscular diseases like ALS, SMA, and muscular dystrophy exercise safely. Because these conditions weaken the nerves that control muscles, traditional exercise is often too hard. W…

This study tests a non-invasive device that gently stimulates a nerve in the ear to see if it can reduce pain and improve daily life for people with post-herpetic neuralgia (long-term nerve pain after shingles). About 34 adults aged 35-65 with persistent pain will receive the sti…

This study tests whether wearing Boarding Ring glasses can help people with vestibular neuritis (a sudden, severe vertigo attack) recover their balance more quickly. Researchers will measure changes in balance and eye movements over one week in 51 adult patients. The glasses are …

This early study tests whether a device that sends electrical pulses to muscles and the spinal cord can improve arm, hand, or leg movement in people with paralysis from spinal cord injury, stroke, or other nerve damage. Up to 64 participants will attend sessions five times a week…

This study tests whether mirogabalin can reduce nerve pain in people with diabetes. 78 adults will take either mirogabalin or the standard drug pregabalin daily for 8 weeks. Researchers will measure pain levels, side effects, and quality of life to see if mirogabalin is a better …

This study tests whether daily symptom reporting via an app, website, or phone, plus follow-up calls from a nurse practitioner, can better manage chemotherapy-induced peripheral neuropathy (nerve damage). 422 adults who have completed certain chemotherapies will track symptoms fo…

This study compares two ways to block pain during lung surgery (VATS) to see if they can prevent chronic pain that lasts for months. Forty adults having planned lung surgery will receive either an ultrasound-guided nerve block or a surgeon-given block. Researchers will track pain…

This study tests whether virtual reality exercises can help people with balance problems caused by a weak inner ear on one side. 48 adults will be split into three groups: one doing standard balance exercises, one using VR, and one doing both. The goal is to see if VR can reduce …

This study tests whether a combination of sensory-motor exercises (like pelvic rocking and strength training) can reduce pain and improve balance in postmenopausal women with polyneuropathy. One hundred women with type 2 diabetes and numbness in their arms and legs will be random…

This study compares a high-tech rehabilitation program—using robotic systems, virtual reality, and balance platforms—to standard physical therapy for people with acute neuromuscular diseases like Guillain-Barré syndrome. About 124 adults aged 18 to 80 will participate. The goal i…

This study looks at whether high-frequency spinal cord stimulation can reduce chronic neuropathic pain in people who have had a brachial plexus avulsion injury. Researchers will track pain levels and daily function in 20 adults who are already scheduled to receive the implant. Th…

This study looks at whether the Alexander technique, a method that teaches better posture and movement habits, can help children aged 5 to 9 with Duchenne muscular dystrophy control their upper limbs and feel better day-to-day. About 33 kids will take part, and researchers will m…

This phase 2 trial is testing whether cannabis-based medicines—THC, CBD, or a combination—can safely reduce chronic nerve pain in Veterans. 320 participants will receive one of these treatments or a placebo for six weeks. The goal is to see if these options offer better pain reli…

This study explores whether acupuncture can reduce nerve pain and numbness caused by chemotherapy in people with gynecologic, urologic, or rectal cancers. About 75 participants will be randomly assigned to receive real acupuncture, sham acupuncture, or usual care for 6 weeks. The…

This study compares two treatments for long-lasting nerve pain after shingles (postherpetic neuralgia). Researchers will give 50 adults either a nerve block or a radiofrequency treatment and measure pain levels. The goal is to find which option works better for easing this stubbo…

This study tests whether a high-dose capsaicin patch (made from chili pepper extract) can reduce chronic tailbone pain (coccygodynia) better than a low-dose patch. About 188 adults with pain lasting over 3 months will receive either the 8% patch or a control patch applied once fo…

This study compares two treatments for mild to moderate carpal tunnel syndrome: osteopathic manipulation (hands-on therapy) and wearing a wrist brace at night. Ten adults diagnosed with carpal tunnel will be randomly assigned to one of the two groups. Researchers will use MRI sca…

This study tests whether immersive virtual reality (IVR) can reduce persistent shortness of breath in people with ALS who already use a breathing machine (non-invasive ventilation). 35 participants will try both a VR session based on medical hypnosis and a music therapy session. …

Diabetes patients often suffer from dry mouth, which affects eating, speaking, and quality of life. This study tests whether endocrinologists can deliver simple oral care—like screening, counseling, and saliva substitutes—during routine diabetes visits. Researchers will follow 80…

This study tests whether a painless brain stimulation technique (tDCS plus ultrasound) can reduce chronic pain in people with carpal tunnel syndrome. About 95 adults with long-term wrist pain will receive either real or fake (sham) stimulation to see if it lowers their pain score…

This study in Ukraine compares two minimally invasive procedures—cryoablation (freezing) and radiofrequency ablation (heating)—to treat nerve pain caused by neuromas from combat injuries. Fifty adults with moderate to severe pain will be randomly assigned to one treatment. The go…

This study looks at whether a surgery called RPNI can reduce nerve pain in people who have had a leg amputated. The surgery takes the cut nerve end and wraps it in a small piece of the patient's own muscle to help it heal. Researchers will follow 20 adults for 24 months to see if…

This study tests whether high-intensity laser therapy (HILT) can reduce pain and improve hand function in people with mild-to-moderate carpal tunnel syndrome. Eighty adults will receive either real or sham laser treatment, plus a wrist splint. Researchers will measure pain, nerve…

This study tests a 12-week supervised rehabilitation program for people with two rare genetic conditions that cause walking and balance problems (spastic ataxias). The program includes twice-weekly therapy sessions and once-weekly pool exercises. Researchers want to see if it red…

This study tests whether a simple breathing exercise device can help people with ALS maintain the strength of their inspiratory muscles—the muscles that pull air into the lungs. Over 44 participants will do resisted breathing exercises twice daily for 12 weeks, then twice weekly …

This study looks at whether adding neck and upper back exercises to standard physical therapy can reduce pain and improve hand function in people with carpal tunnel syndrome. Sixty adults with the condition will be split into two groups: one gets standard therapy alone, the other…

This study tests if a non-invasive device that stimulates the vagus nerve can reduce pain, numbness, and tingling in people with diabetic nerve damage. Researchers will measure pain levels, nerve signals, and walking ability in 40 adults aged 45-60. The goal is to find a drug-fre…

This study tests whether giving a mild electrical stimulation to the nerve a week before surgery can improve hand function recovery in people with severe cubital tunnel syndrome. Thirty adults with severe nerve damage will be randomly assigned to receive either real stimulation o…

This study tests whether a noninvasive procedure called transcutaneous pulsed radiofrequency can reduce chronic pain from postherpetic neuralgia, a complication of shingles. The procedure delivers electrical pulses through electrodes placed on the skin over the painful area. Rese…

This study compares a continuous cooling therapy device to traditional ice packs for managing pain after open carpal tunnel release surgery. About 128 adults having the surgery will be randomly assigned to one of the two cooling methods. Researchers will measure pain levels and h…

This study tests whether a spinal cord stimulator—a device that sends mild electrical pulses to the spine—can reduce pain and improve blood flow in the legs of people with type 2 diabetes who have nerve pain. Researchers will enroll 25 adults with painful diabetic neuropathy and …

This study uses special PET scans to measure inflammation in the brains of people with Alzheimer's, Parkinson's, ALS, and other neurodegenerative diseases. Researchers will compare these scans to those from healthy volunteers to see if inflammation levels differ. The goal is to d…

This study at the National Institutes of Health looks at how infections can worsen symptoms in people with mitochondrial disease, a group of disorders that affect energy production in cells. Researchers will evaluate participants' immune systems through blood tests, physical exam…

This study follows 2,800 unpaid caregivers of people with chronic conditions like diabetes or inherited disorders. Researchers will survey and interview caregivers over up to 5 years to track changes in their health, stress, and social support. The goal is to understand how careg…

This registry study follows adults with myasthenia gravis (MG) over time to understand how symptoms, daily functioning, and quality of life change. Participants complete surveys about their symptoms, treatments, and challenges, and may share medical records. The goal is to gather…

This study aims to understand why some people are more prone to fungal infections, especially from Candida yeast. Researchers will collect blood, saliva, urine, and tissue samples from people with immune disorders, their family members, and healthy volunteers. No treatment is giv…

This study looks at whether liver function and blood clotting tests can help doctors better understand and monitor cardiac transthyretin amyloidosis (ATTR-CA), a condition where abnormal protein builds up in the heart. Researchers will compare these tests in people with ATTR-CA a…

This study tests whether simple reminders—called nudges—sent to doctors and patients can increase the use of genetic testing for people with certain conditions like Parkinson's disease, ALS, or heart problems. The trial will compare different ways of prompting genetic testing, su…

This study follows 75 people aged 6 months or older with congenital myasthenic syndromes (CMS), a group of inherited disorders that weaken communication between nerves and muscles. Over up to 7 visits in 5 years, researchers will use physical exams, heart and lung tests, MRIs, an…

This observational study is looking at men with spinal and bulbar muscular atrophy (SBMA), a rare inherited disease that causes gradual muscle weakness. Researchers will track changes in muscle strength, imaging scans, and blood or spinal fluid markers over two years. The goal is…

This study screens 360 adults with neurodegenerative disorders like ALS, frontotemporal dementia, and progressive supranuclear palsy. Researchers will use medical history, physical exams, memory tests, movement analysis, MRI scans, and other tests to find common features and diff…

This study investigates whether people who had carpal tunnel surgery might have a higher chance of having ATTR amyloidosis, a condition where abnormal proteins build up in tissues, potentially affecting the heart. Researchers will biopsy tissue from about 180 patients who had car…

This study gathers medical information from 200 adults with possible nerve, muscle, or autonomic nervous system disorders who are not already in other NIH studies. Participants undergo standard tests like nerve conduction studies, EMG, and physical exams. The data is stored anony…

This study follows children with congenital or childhood myotonic dystrophy over time to see how their symptoms change. Researchers will test a range of assessments, from walking speed and hand strength to language and quality of life, to find the best ways to measure the disease…

This study aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…

This study is building a large online registry of US adults who experience sinus, ear, dizziness, and headache symptoms, with or without a migraine diagnosis. Participants answer questions about their symptoms, how these affect daily life, and which treatments they have tried. Th…

This study creates a registry to collect medical information from people with rhizomelic chondrodysplasia punctata (RCDP) and closely related conditions. The goal is to better understand the natural history of these rare disorders and identify factors that may predict health outc…

This study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …

This study investigates how engaging in structured conversations affects the speech of people with amyotrophic lateral sclerosis (ALS) compared to typical speakers. Participants will complete speaking tasks both alone and with a partner, allowing researchers to measure speech cla…

This study looks at how the severity of carpal tunnel syndrome relates to ultrasound images of the nerve, nerve conduction tests, hand grip strength, and pain sensitivity. Researchers will assess 90 adults with the condition using these measures during a single visit. The goal is…

This study is looking for biological clues in the blood that explain why many people with ALS lose weight. Researchers will follow 1,000 patients in France and Germany, collecting blood samples and questionnaires alongside standard care. The goal is to find markers that could lea…

This study follows 1,000 adults with myotonic dystrophy type 1 (DM1) over time to measure how the disease progresses. Researchers will track walking speed, hand muscle relaxation, grip strength, heart and lung function, and daily activity. The goal is to establish reliable biomar…

This study is a large registry that collects health and genetic information from people with a personal or family history of pancreatic tumors. The goal is to find the genes and other factors that cause pancreatic cancer to run in some families. By studying thousands of participa…

This study will observe 30 children with type I or type II spinal muscle atrophy who are taking risdiplam. Researchers will track motor function improvements over 24 months using standard tests. The goal is to see how well the drug works in everyday medical practice.

This study is testing a new, painless device called electrical impedance myography (EIM) to measure muscle health. Researchers will use it on healthy volunteers and people with neuromuscular diseases to see how well it works compared to standard tests like ultrasound and nerve st…

This study follows people with SPLIS, a rare inherited metabolic condition, to map its natural history—how the disease unfolds over time. Researchers will track growth, organ function, quality of life, and survival in about 28 participants. The goal is to understand the full rang…

This study looks at whether levels of a protein called HMGB1 are different in people with LAMA2-related muscular dystrophy, a condition that causes severe muscle weakness. Researchers will measure HMGB1 in blood and muscle samples already collected from about 25 patients. The goa…

This study follows people with several types of spinocerebellar ataxia (SCA) — rare genetic diseases that cause balance, coordination, and speech problems — to learn how these conditions change over time. Researchers will collect blood samples, perform neurological exams, and use…

This study is for people with ALS and healthy volunteers. It uses brain and spine MRI scans and blood tests to find markers that show how the disease progresses. The goal is to develop better tools to measure ALS over time. About 90 participants will be followed for up to one yea…

This study tests whether a non-invasive electrical test on the quadriceps (thigh muscle) can help diagnose and monitor amyotrophic lateral sclerosis (ALS). Researchers will measure nerve signals traveling from the brain to the muscle in 30 people with suspected or confirmed ALS. …

This long-term study follows people with or at genetic risk for frontotemporal lobar degeneration (FTLD), a group of brain disorders that cause changes in behavior, language, and movement. Researchers will use brain scans, memory tests, and blood samples to measure how the diseas…

This study aims to discover biological markers (biomarkers) that could help doctors diagnose and track neurological autoimmune diseases such as multiple sclerosis, myasthenia gravis, and Guillain-Barre syndrome. Researchers will analyze samples from 50,000 people, including patie…

This study investigates how balance and cognitive tasks—like walking while doing mental math—affect each other in older adults. Researchers will compare healthy seniors with those who have experienced dizziness or falls, using wearable sensors and cognitive tests. The goal is to …

This early-stage trial tests the safety and tolerability of a single dose of AH-008, a potential treatment for chemotherapy-induced peripheral neuropathy (nerve damage). Healthy adults aged 18 to 65 receive either AH-008 or a placebo by intravenous infusion. The study monitors si…

This study aims to understand how heart problems develop in people with Duchenne and Becker muscular dystrophy, as well as in carriers. Researchers will collect cardiac MRI scans and clinical data from 1,000 participants to build a registry. Using advanced image analysis and deep…

This study aims to create a simple, objective test to detect nerve damage caused by the chemotherapy drug vincristine in children and teens. Researchers will use a new device to measure nerve function and compare it to standard assessments. The goal is to help doctors adjust chem…

This observational study follows about 200 people in the United States who have chronic inflammatory demyelinating polyneuropathy (CIDP) and still experience disability despite at least three months of standard treatment. Researchers will track changes in disability scores and ex…

This international online study collects medical, behavioral, and developmental information from people with rare genetic changes that are linked to autism and other neurodevelopmental disorders. By partnering with families, researchers aim to build a detailed database to improve…

This study builds a global database of people diagnosed with or at risk for Krabbe disease, a rare nerve disorder. Researchers will track symptoms, genetic mutations, and enzyme levels to find clues that predict when the disease starts and how severe it becomes. The registry also…

This study looks at three common hand function tests — the Jebsen-Taylor Hand Function Test, the Minnesota Dexterity Test, and the Nine-Hole Peg Test — to see how reliable and accurate they are for people with carpal tunnel syndrome. About 100 participants with the condition will…

This study compares walking patterns between people with diabetic peripheral neuropathy (nerve damage from diabetes) and healthy adults. It also tests whether giving people real-time feedback on their walking form can immediately change how they walk. The goal is to understand th…

This study is testing whether a non-invasive nerve monitoring device called TetraGraph can accurately detect early signs of ICU-acquired weakness. Researchers will compare readings from the device with standard needle-based electromyography (EMG) tests in healthy volunteers and I…

This study looks at 70 adults with sensory neuronopathy, a rare nerve condition that causes balance and coordination problems. Researchers will test several clinical scales over 12 months to see which one best tracks how the disease changes over time. The goal is to find a reliab…

This study examines whether three specific signs seen on CT scans — nerve-root swelling, tissue infiltration around the nerve, and increased density of the nerve — can identify the exact nerve root causing sciatica. Researchers reviewed planning CT scans from 400 adults with low-…

This study investigates whether a blood test can predict which stomach cancer patients will develop nerve damage (peripheral neuropathy) from the chemotherapy drug paclitaxel. Paclitaxel is a standard second-line treatment for advanced gastric cancer, but nerve damage is a common…

This study creates a biobank to collect and store tissue samples—such as blood, muscle, saliva, urine, and stool—from people with neuromuscular diseases, their family members, and healthy volunteers. The samples will be shared with researchers to help them better understand these…

This study will check if certain blood markers can predict how well a nerve block procedure works for people with nerve pain. Researchers will measure pain levels and blood inflammation markers before and after the procedure in 120 adults. The goal is to see if these blood tests …

This study looks at how the location of brain-stimulating electrodes affects pain relief in adults with long-term nerve pain that hasn't improved with other treatments. Researchers will use brain scans to see which areas are activated and how that relates to pain reduction. The g…

This study follows 250 people who had ankle fractures to see how many develop nerve-related chronic pain after the bone heals. Participants answer pain questionnaires and undergo simple sensory tests. The goal is to better understand this type of pain, not to test any new treatme…

This study is looking for early signs of heart amyloidosis in Black adults who carry a specific gene change (V122I TTR) that raises their risk. Researchers will use heart MRI scans and blood tests to detect protein buildup before symptoms appear. The goal is to find ways to diagn…

This study looks at whether certain proteins in the blood can help doctors monitor severe traumatic brain injury and predict who might benefit from hyperbaric oxygen therapy. Researchers will analyze blood samples from 150 patients already enrolled in a larger trial testing diffe…

This study follows 50 children who had a nerve-freezing procedure (cryoneurolysis) before surgery to correct a sunken chest (pectus excavatum). Researchers check for long-term nerve pain, quality of life, and sensory recovery using questionnaires. The goal is to see if the pain r…

This study is translating a questionnaire about brachial plexus birth palsy into Turkish and checking if it works well. 50 adults with this condition will fill out the new Turkish version along with other standard surveys. The goal is to provide a reliable tool for Turkish-speaki…

This study compares carbon dioxide emissions from orthopedic surgeries performed with different materials and setups. Researchers will track waste, energy use, and sterilization methods for procedures like carpal tunnel release or wound revision. The goal is to identify ways to r…

This study looks at how different mutations in the MORC2 gene lead to different symptoms, from Charcot-Marie-Tooth disease to a more complex condition called DIFGAN. Researchers will collect skin and blood samples from 45 participants to measure epigenetic and genetic markers. Th…

This study follows adults who had shoulder tendon transfer surgery as children for obstetric brachial plexus palsy, a nerve injury during birth. Researchers will measure pain, arm movement, strength, and social appearance anxiety in 80 participants and compare them to healthy pee…

This study is looking at whether people with a common inner-ear condition called benign paroxysmal positional vertigo (BPPV) have lower vitamin D levels than those without it. Researchers will compare vitamin D in 160 adults — half with BPPV and half without. The goal is to see i…

This study follows 60 people with ALS to see how swallowing problems change over time and how they affect quality of life. Researchers also want to understand patients' feelings about getting a feeding tube (PEG). The goal is to learn more, not to test a new treatment.

This study checks if a French version of the MEPS/MEPI elbow score works well for children aged 4 to 17. It includes kids with and without elbow injuries to see if the score accurately measures elbow pain and function. The goal is to make sure doctors can use this tool reliably i…

This study looks at people with cavovarus foot deformity (a complex foot shape) due to Charcot-Marie-Tooth disease. Researchers want to see if a simple physical exam can identify different foot types, which currently requires special software. If successful, this could help surge…

This study is following 700 adults with myotonic dystrophy type 1 (DM1) over two years. Researchers are measuring walking speed, lung function, and muscle tissue changes to find reliable markers of disease progression. The goal is to improve future clinical trials by better under…

This study is for children up to 4 years old with congenital myotonic dystrophy type 1 (CDM). Researchers want to find better ways to measure how the disease affects motor skills and language, and to identify biological markers. The goal is to improve future clinical trials and c…

This study is testing whether a watch-like device worn on the ankle can measure how well toddlers with Duchenne muscular dystrophy (DMD) move during their daily activities. Researchers will ask 30 boys with DMD and 30 without (ages 1-3) to wear the device for three 28-day periods…

This study aims to collect DNA samples and detailed eye exam data from 1,000 people with rare inherited eye diseases like aniridia, Best disease, and albinism. Participants provide a saliva or blood sample and share their eye health records. The goal is to expand a research repos…

This study tracks quality of life in 200 adults with chronic respiratory failure who use breathing support like non-invasive ventilation. Researchers will use questionnaires to see how treatments impact daily living over time. The goal is to better understand patient experiences,…

This study looks at how to best use a machine that helps people with neuromuscular disease cough and clear mucus from their lungs. Researchers will test different pressure settings to see which works best without causing side effects. About 30 adults with stable or slowly progres…

This study gathers medical information from people with MNGIE, a rare genetic disease that affects the nerves and digestive system. Researchers want to learn how the disease progresses and find better ways to measure it. Up to 50 patients worldwide can join, and no new treatments…

This study looks at how the FDA-approved drug edaravone (Radicava) affects people with different forms of ALS. Researchers will collect blood, urine, and spinal fluid samples from 160 participants to measure markers of oxidative stress. The goal is to identify which ALS patients …

This study aims to learn why myotonic dystrophy type 1 affects children differently than adults, and why symptoms vary even within the same family. Researchers will observe 100 children (ages 0-17) through video calls and simple at-home activities, and analyze their genes from a …

This study looks at whether a vestibular implant (a device that helps with balance) can improve how people with severe inner ear damage perform tasks like staying upright, navigating, and thinking. About 20 people who already have the implant will be tested with it on and off, an…

This study looks at how spinal cord stimulation (SCS) affects brain activity in veterans with long-term nerve pain. Researchers will use non-invasive brain scans (fNIRS and EEG) to compare brain connectivity in SCS users and those being evaluated for SCS. The goal is to understan…

This study looks at how inhaled cannabis affects the brain in people with chronic low back pain. Researchers will use MRI scans to measure changes in brain blood flow and oxygen levels, and ask participants to rate their pain. The goal is to understand the brain mechanisms behind…

This study tests whether a playful coaching feature on a cough-assist device helps children learn to use it more effectively. Researchers will compare learning speed and cough quality in healthy children aged 6-12, and later in children with neuromuscular diseases. The goal is to…

This study looks at how the drug enfortumab vedotin affects nerves in people with advanced or muscle-invasive bladder cancer. Researchers will use nerve tests and patient surveys to track changes over 9-15 weeks. The goal is to better understand and potentially reduce nerve damag…

This study will collect electronic health records from up to 2500 people with Duchenne or Becker muscular dystrophy, including female carriers, across U.S. clinics. The data will be combined with patient-reported information to give researchers a fuller picture of the diseases. T…

This study collects blood, skin, and spinal fluid samples along with health information from 2,500 adults with neuromuscular diseases like ALS, neuropathy, and muscular dystrophy. The goal is to store these samples and share them with researchers to speed up discoveries about how…

This study uses three wearable devices—a continuous glucose monitor, a heart monitor, and an activity tracker—to see if high blood sugar is linked to heart problems in people with Duchenne muscular dystrophy. Researchers will compare 80 participants with and without DMD. The goal…

This study looks at people with long-term nerve pain caused by taxane chemotherapy. It compares two groups: one using capsaicin 8% patches (made from chili peppers) and another receiving standard pain treatments. The goal is to see if the patches help small nerve fibers in the fe…

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This study aims to find genetic causes and risk factors for ALS by analyzing DNA from 1,200 people in Norway with probable or definite ALS. Researchers will look for known disease-causing mutations and search for new genes linked to the disease. The goal is to better understand w…

This study looks at whether changes in smell and taste contribute to poor nutrition in people with ALS. Researchers will measure food preferences in 60 adults with ALS using a computer test. The goal is to find new ways to improve diet and quality of life by personalizing food ad…

This study tests whether non-invasive sensors can detect reduced blood flow in the legs during long pelvic surgeries. Thirty adults will be monitored during their procedure. The goal is to see if this approach can help prevent complications like limb ischemia.

This study follows 100 adults who had surgery for traumatic nerve injuries in the hand or arm. Researchers will measure hand function, strength, and quality of life over time to better understand long-term recovery. The goal is to gather information, not to test a new treatment.

This study looks at how tired people with spinal muscular atrophy (SMA) feel during daily activities while taking the drug nusinersen. Researchers will use a new questionnaire called the SMA EFFORT to measure this fatigue. The goal is to better understand fatigue in SMA and see i…

This study follows up to 60 babies with spinal muscular atrophy (SMA) found at birth, plus 30 healthy babies, for up to 30 months. Researchers use wearable devices worn at home to track muscle development and find when movements start to differ from normal. The goal is to identif…

This study follows 450 people with FSHD for at least 3 years to measure how the disease affects walking, arm use, breathing, and other daily activities. The goal is to improve patient care and help researchers design better clinical trials by understanding what changes in movemen…

This study is building a worldwide online registry for people with Duchenne or Becker muscular dystrophy, including female carriers. Participants share their health information to help researchers learn more about the disease and to match patients with clinical trials. The goal i…

This study looks at a specific gene (IGLV1-44) to understand why it causes two different diseases: AL amyloidosis and POEMS syndrome. Researchers will analyze blood and bone marrow samples from 100 adults with these conditions or multiple myeloma. The goal is to find genetic patt…

This study looks at tremor (shaking) in people with Charcot-Marie-Tooth (CMT) disease, a nerve condition. Researchers will use muscle and movement tests to better understand why tremor happens and how it affects daily life. About 75 adults with CMT will take part, including some …

This study looks at how certain substances applied to the skin affect blood flow in people with nerve pain caused by chemotherapy. Researchers will compare healthy volunteers and patients to better understand the pain. The goal is to learn more about the condition, not to test a …

This study is developing a standard test to measure how well assistive technologies (like eye trackers) and brain-computer interfaces help people with spinal cord injury or ALS use computers and phones. Researchers will test 60 participants, including healthy volunteers and peopl…

This study looks at how muscles grow and change in children with cerebral palsy (CP) by examining tiny samples from leg muscles. Researchers will compare these microscopic findings with muscle images and movement tests. The goal is to better understand why muscles in CP develop d…

This study is testing a new, non-invasive brain monitor (Neurosteer) in 100 ICU patients who are on breathing machines and sedatives. The monitor is a small device placed on the forehead that measures brain activity. Researchers want to see if it can accurately track levels of se…

This study gathers medical information from 200 people with glycogen branching enzyme deficiency, known as GSD IV or APBD. The goal is to learn how these rare diseases progress over time. No new treatments are tested; instead, researchers review clinic records to better understan…

This study is testing a new way to measure fatigue in people with spinal muscular atrophy (SMA), a condition that causes muscle weakness. The test is designed to be easy enough for patients with different levels of ability. Researchers want to see if the test is safe, doable, and…

This early study tests whether a brain implant can help people with severe paralysis from conditions like ALS, spinal cord injury, or stroke control devices using their thoughts. The implant records brain signals to decode text or synthesized speech. Only 3 adults will participat…

This study looks at how often people with Charcot-Marie-Tooth disease (CMT) have problems with their autonomic nervous system, which controls things like heart rate and sweating. Researchers will use questionnaires and simple, non-invasive tests to measure these symptoms in 50 ad…

This study explores whether a non-invasive brain-computer interface (BCI) can help people with motor disorders, such as spinal cord injury or stroke, control assistive devices using their thoughts. Researchers will record brain signals with EEG and use machine learning to interpr…

This study is testing whether tiny particles in the blood, called extracellular vesicles, can predict nerve damage caused by chemotherapy. Researchers will follow 120 adults with various cancers who are receiving specific chemo drugs. Participants give blood samples and answer qu…

This study is building a large registry of people with ALS in the United States. The goal is to count how many people have the disease and learn about possible causes, such as environmental or genetic factors. Anyone 18 or older with ALS can join. The registry does not test a tre…

This study looks at how nerves are affected in people who develop nerve damage from the chemotherapy drug cisplatin. Researchers will use a special test to measure nerve activity and compare it to normal nerves. The goal is to better understand this side effect and find early war…

This study looks at whether a common heart procedure (using the wrist artery) can damage nerves in the hand. Researchers will check 400 patients for signs of carpal tunnel syndrome or other nerve problems. The goal is to understand the risks better, not to test a new treatment.

This study is testing a device called the mScan to see if it can track how ALS progresses over time. Researchers will measure electrical signals in muscles and compare them to standard tests. The goal is to find a simple, objective way to monitor the disease that could be used at…

This study looks back at the last 10 years in Denmark to understand how many people use breathing machines at home through a tube in their windpipe. Researchers will track which diseases lead to this need and how many patients survive one year after starting. The goal is to spot …

This study follows 100 people with ALS to see how their autonomic and sensory nerves change over time. Researchers will use skin biopsies and other tests to track nerve damage and look for biomarkers that could help predict disease progression. The goal is to better understand AL…

This study is looking at whether people with benign paroxysmal positional vertigo (BPPV) have differences in their autonomic nervous system compared to healthy people. Researchers will measure heart rate variability (HRV) in 104 participants (52 with BPPV and 52 matched controls)…

This study looks at people with shoulder pain lasting at least 3 months to understand if nerve-related pain and a condition called central sensitization (where the nervous system becomes overly sensitive) play a role. Researchers will use questionnaires and simple movement tests—…

This study aims to find early biological markers (biomarkers) for Charcot-Marie-Tooth disease type 1A (CMT1A), a common inherited nerve disorder. Researchers will analyze blood and nerve samples from 70 people aged 10-30 with CMT1A, along with animal models, to identify signs tha…

This study takes skin biopsies and blood samples from people with ALS, healthy carriers of ALS-related gene mutations, and control subjects. Researchers will grow skin cells in the lab to study how the disease works at a cellular level. The goal is to better understand ALS, not t…

CAPTURE ALS is a long-term study that collects health data and biological samples from 150 people with ALS and related conditions. The goal is to create a shared resource for researchers worldwide to better understand the disease and develop new treatments. No experimental drugs …

This study watches how children under 5 with LAMA2-related muscular dystrophy move and develop over two years. Researchers will test muscle function, swallowing, breathing, and collect blood samples. The goal is to learn more about the disease so future treatments can be tested e…

This study aims to uncover genetic and biological differences between people with Menière's disease, those with vestibular migraine, and healthy individuals. Researchers will analyze blood samples from 250 participants to look for specific gene variations and biohumoral factors. …

This study is testing whether special insoles worn inside shoes can measure how well children with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk. About 106 participants, including healthy children, will wear the insoles during walking tests and in daily …

This observational study follows 75 people with shoulder impingement syndrome who are already receiving standard physical therapy. Researchers want to see if nerve-related pain and negative thinking about pain (called pain catastrophizing) affect how well patients stick to their …

This study aims to understand how different types of changes in the DMD gene are linked to heart problems in boys and young men with Duchenne or Becker muscular dystrophy. Researchers will use heart tests and blood samples to look for early signs of heart issues. The study involv…

This study follows nearly 3,000 people, including healthy adults and those with memory or movement problems, to learn how brain diseases like Alzheimer's and Parkinson's develop. Researchers use advanced brain scans and tests to track changes over time. The goal is to improve ear…

This study looks at how people with ALS and their caregivers communicate verbally. Researchers will record conversations and measure speech clarity, sentence complexity, and pauses. The goal is to understand how communication changes over time. 152 participants will take part in …

This study uses a new ultra-high-resolution PET scanner to take detailed pictures of the brain and spinal cord in 300 healthy volunteers and people with conditions like Alzheimer's, Parkinson's, ALS, and psychotic disorders. The goal is to understand how small brain regions chang…

This study looks at 57 people aged 20 to 70 who carry the Val50Met gene variant linked to hereditary ATTR amyloidosis, a condition that can damage nerves and the heart. Researchers want to describe the different symptoms people experience, such as heart problems, nerve issues, or…

This study is gathering information from 4500 people who have or will have brain surgery for conditions like aneurysms, tumors, epilepsy, and Parkinson's disease. The goal is to find patterns that help doctors predict how well someone will recover. By standardizing how outcomes a…

This study aims to find biological markers (biosignatures) in people with ALS and FTD to make diagnosis more precise. Researchers will analyze samples like spinal fluid, skin, and tears from 230 participants using advanced techniques. The goal is to create a 'disease fingerprint'…

This study aims to find early markers of Parkinson's disease, multiple system atrophy, and Lewy body dementia by analyzing samples from the nose, blood, and urine. Researchers will compare results from 180 people with these conditions and healthy volunteers. The goal is to improv…

This study aims to understand why myotonic dystrophy type 1 affects people so differently. Researchers will remotely assess muscle strength, memory, and activity in 1,000 participants, and analyze their DNA from a blood sample. All activities are done from home using a mailed too…

This study looks at how the constant vibration from tattoo machines may damage nerves in the hands of tattoo artists. Researchers will test touch, strength, and sensation in 20 tattoo artists and compare them to 20 people who don't use vibrating tools. The goal is to understand i…

This study is creating a large database of 1,000 people with ATTR amyloidosis, a rare disease that causes abnormal protein buildup in organs. Researchers will collect health information over time to better understand how the disease progresses and to develop tools that help docto…

This study is creating a detailed registry of 500 people with hereditary spastic paraplegia (HSP), a rare disorder that causes progressive leg stiffness and weakness. Researchers will collect medical history, genetic data, and patient feedback over time to better understand how t…

This study examines DNA from 575 breast cancer patients who received the chemotherapy drug paclitaxel. Researchers want to find genetic differences that explain why some patients develop nerve damage (peripheral neuropathy) while others do not. The goal is to identify biomarkers …

This study looks at how physical abilities impact the quality of life of people with ALS, a disease that weakens muscles over time. About 30 adults with ALS will fill out questionnaires about their daily function and well-being during regular clinic visits for up to two years. Th…

This study explores whether small, skin-friendly sensors can detect early signs of muscle tone problems in newborns up to 8 weeks old. Researchers will compare sensor data with standard medical exams to see if the technology can identify atypical muscle development sooner. The go…

This research study at Boston Children's Hospital is looking at the genes and proteins involved in congenital myopathies—rare muscle diseases that are present from birth. Researchers will analyze DNA from up to 4,000 participants, including patients and their family members, to f…

This study looks at how common nerve pain is in people aged 80 and older. Researchers will check pain levels and overall health in 450 volunteers. The goal is to better understand the link between nerve pain and aging, not to test a new treatment.

This study, called CRiALS, is a large umbrella protocol that aims to recruit up to 10,000 people. It includes individuals with ALS or related neurodegenerative diseases, their family members, and healthy volunteers. The main goal is to build a pool of participants for a variety o…

This study follows healthy people who have a family history and genetic risk for ALS. Researchers want to find early warning signs and understand what triggers the disease. Participants do not receive any treatment, but their health is tracked over time. The goal is to learn how …

This study aims to uncover the genetic causes of hereditary spastic paraplegia (HSP), a group of rare neurological diseases that cause progressive muscle stiffness and weakness. Researchers at Boston Children's Hospital will analyze DNA from 200 people with HSP to find genetic va…

This study is looking at whether the way people with spinal muscular atrophy (SMA) walk can tell us about their bone health. Researchers will use special insoles to measure walking patterns and compare them with bone density scans and blood tests. The goal is to find simple, non-…

This study is collecting health data and blood samples from 100 people with specific types of hereditary spastic paraplegia (SPG4 and SPG5A). The goal is to create a shared database and biobank to better understand how the disease progresses. This information will help prepare fo…

This study tests two ways of cycling while lying in a hospital bed for patients in the intensive care unit (ICU). One method is steady cycling for 10-20 minutes, and the other is short bursts of harder cycling mixed with easier pedaling. The goal is to see which approach better a…

This study collects health information and biological samples from up to 700 people under 30 with early-onset hereditary spastic paraplegia (HSP). Researchers aim to better understand how the disease progresses over time and create a registry for future studies. Participants prov…

This study looks at how changes in muscles and the brain are linked in aging and diseases like ALS, Alzheimer's, and Parkinson's. Researchers will compare healthy older adults with those who have these conditions to find early signs of disease. The goal is to better understand ri…

This study investigates whether serious muscle weakness, known to affect ICU patients, also occurs in people hospitalized outside the ICU. Researchers will follow about 600 adults in Italian hospitals, half with organ failure and half without, to see how often weakness develops a…

This study aims to translate and test the Situational Vertigo Questionnaire (SVQ) in Turkish. The SVQ measures dizziness triggered by visual environments like supermarkets or driving. Researchers will check if the Turkish version is valid and reliable by comparing it with other d…

This study tests whether real-time visual and audio cues can help people with diabetic peripheral neuropathy walk better. Researchers will measure foot pressure and push-off force as 25 participants walk on a treadmill. The goal is to understand how biofeedback changes walking pa…

This study follows 180 children in Italy born with biotinidase deficiency, a rare condition that can cause skin, eye, and nerve problems if untreated. Researchers will compare each child's genetic makeup with their enzyme activity levels and health outcomes. The goal is to better…

This study follows 75 adults with CMT1A and 25 healthy volunteers over five years to track how the disease progresses. Researchers will analyze blood and skin samples to find biomarkers that could make future clinical trials faster and more reliable. The goal is to better underst…

This study looks at how nerves work in people with Charcot-Marie-Tooth disease and other nerve disorders like CIDP. Researchers will use special electrical tests and ultrasound to measure nerve and muscle changes. The goal is to better understand these conditions, not to test a n…

This study investigates whether brain surface recordings can decode Mandarin Chinese speech, including tones, from imagined speech. About 50 participants with brain tumors or epilepsy will have temporary electrodes placed during surgery, and 10 people with severe speech loss will…

This study looks at immune cells called macrophages in people with ALS, a disease that affects movement. Researchers will compare these cells between ALS patients, healthy people, and those with other motor problems. The goal is to find new targets for future treatments and bette…

This study aims to identify genetic factors that affect how severe muscle and heart problems become in people with LMNA gene mutations. Researchers will collect skin and muscle samples from 40 participants and use advanced DNA and RNA analysis to look for protective or aggravatin…

This study looks at how type 1 diabetes affects the nerves that control the heart in children. Researchers will measure heart rate variability in 100 children with diabetes and compare them to healthy kids of the same age and sex. The goal is to find early signs of cardiac dysaut…

This study follows children and adults with Tay-Sachs, Sandhoff, and GM1 gangliosidosis to understand how these diseases progress. Researchers will measure changes in motor skills, communication, and behavior each year. The goal is to create a clear picture of the diseases so fut…

This study tests a new training method for caregivers of infants with brachial plexus birth injury (a nerve injury that can cause arm weakness). The goal is to see if the training helps caregivers feel more confident and do daily stretching exercises more consistently, which may …

This study looks at whether quick, easy walking tests can replace the current complex and time-consuming exam used to track walking problems in people with facioscapulohumeral muscular dystrophy (FSH). Researchers will compare results from simple tests like the 6-minute walk with…

This study is collecting information from 4,000 adults with peripheral neuropathy (nerve damage) to better understand the condition. Researchers will combine standard tests with nerve ultrasound to see if it helps diagnose and track the disease. No new treatments are being tested…

This study looks at children and teens who had a limb removed before age 15-16 to see if they experience nerve pain (phantom limb or stump pain). Researchers will review medical records and use questionnaires during regular check-ups. The goal is to better understand how common t…

This study is testing different ways to measure muscle strength and movement in children with a rare muscle disease called FSHD. The goal is to find the best tests to use in future studies of new treatments. About 80 children aged 5 to 17 with confirmed FSHD will take part. The r…

This study is looking at whether a molecule in the blood called miR-1 can help doctors understand how muscle diseases like Duchenne muscular dystrophy and myotonic dystrophy are progressing. Researchers will compare miR-1 levels in 104 people, including patients with different mu…

This study aims to understand how upper-limb assistive devices—like grasping gloves, mealtime aids, and robotic arms—impact the daily lives of people with motor impairments. Researchers will interview and survey 150 regular users (including those with stroke, spinal cord injury, …

This study looks at whether tiny molecules in the blood, called microRNAs, can help predict how often and how severely cancer patients develop nerve damage from chemotherapy. Researchers will study 90 people receiving specific chemo drugs for lung or colon cancer. The goal is to …

This study creates a registry for people with facioscapulohumeral muscular dystrophy (FSHD) to share their health experiences and data. Up to 5,000 participants in the U.S. will provide information through surveys to help researchers better understand the disease and develop impr…

This study is gathering information from 1,500 people with cluster headache or trigeminal neuralgia to learn more about these painful conditions. Researchers will collect surveys, genetic samples, and other health data. The goal is to better understand what causes these headaches…

This study follows 220 boys aged 4 to 9 with Duchenne muscular dystrophy (DMD) for up to 3 years. Researchers collect data on muscle strength, walking ability, heart and lung function, and quality of life to better understand how the disease progresses. The goal is to gather natu…

This study aims to find genes that cause eye misalignment (strabismus) and related conditions involving the cranial nerves and brainstem. Researchers will analyze DNA from up to 20,000 participants with these disorders. The goal is to better understand the genetic causes, which c…

This study looks at whether measuring big toe strength can help doctors understand nerve damage caused by chemotherapy (called CIPN). Researchers will compare 30 people with CIPN to healthy adults using a special device called ToeScale. Participants will also answer questionnaire…

This study follows 1500 people with rare genetic brain disorders to learn how these diseases progress. Researchers measure thinking, movement, and daily living skills over time, and also look at brain scans and body fluids. The goal is to better understand the diseases and how tr…