Neuromuscular disease

This study tests the long-term safety and effectiveness of an experimental drug called BBP-418 (ribitol) in people with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle-weakening disease. Participants who completed a previous study will take BBP-418 orally twice d…

This study tests the long-term safety and effectiveness of an experimental drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). Participants who completed a previous VX-670 study can join. The drug is given through a vein, and researchers will monitor side effects a…

This study tests a gene therapy called PBKR03 for babies with early infantile Krabbe disease, a severe genetic disorder that damages the brain and nerves. The treatment delivers a working copy of the GALC gene to the brain and body. Researchers will check safety and find the best…

This early-stage trial tests a single injection of ARO-ATXN2 in 36 adults with spinocerebellar ataxia type 2, a rare genetic disorder that affects movement and coordination. The main goal is to see if the drug is safe and how the body processes it. Participants are randomly assig…

This phase 2 trial tests whether the experimental drug VHB937 can slow the decline in function and delay the need for breathing support in people with early-stage ALS. About 251 participants will receive either VHB937 or a placebo for 40 weeks, followed by an open-label extension…

This early-stage trial is testing a drug called BMN 351 in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The drug is designed to help the body produce a shorter but still useful version of the muscle protein dystrophin. The main goal is…

This study planned to test whether injecting botulinum toxin (Botox) into the salivary glands could reduce drooling in people with true bulbar palsy, a condition that causes swallowing problems after a brain stem injury. The trial was designed to compare the injections against st…

This phase 3 trial tests whether the drug troriluzole can slow the progression of spinocerebellar ataxia, a rare genetic disorder that affects coordination and balance. About 300 adults with different types of SCA are randomly assigned to take either troriluzole or a placebo dail…

This Phase 3 trial tests whether the oral drug remibrutinib works better than teriflunomide at reducing relapses and slowing disability in people with relapsing multiple sclerosis. About 1,000 adults aged 18–55 with active MS are taking part. The study is double-blind, meaning ne…

This study tests a donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…

This Phase 2 trial tests a drug called RO7204239 in 51 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. The drug is an antibody that blocks myostatin, a protein that normally limits muscle growth, to help pres…

This program allows eligible patients to receive RGX-202, a gene therapy, on a single-patient basis outside of a clinical trial. It is designed for those with serious conditions who have no other treatment options. Currently, the program is temporarily not available.

This clinical trial is testing whether dimethyl fumarate, a drug already used for multiple sclerosis, can improve balance and walking in adults with adrenomyeloneuropathy (AMN), a rare nerve disease. Forty participants will take either the drug or a placebo daily for 36 months, w…

This study tests a gene therapy called vesemnogene lantuparvovec in about 20 people with spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The therapy delivers a working copy of the SMN1 gene to help muscles work better. The main goal is to check saf…

This early-stage trial is testing a drug called NS-050/NCNP-03 in 20 boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The drug is designed to skip a faulty section of the dystrophin gene, allowing the body to produce a shorter but still functional…

This study is a long-term extension of earlier trials testing the drug riliprubart in adults with CIDP, a condition where the immune system attacks the nerves, causing weakness and numbness. About 300 participants who completed a prior riliprubart study will continue receiving th…

This study tests whether adding an experimental drug (RO7204239) to an existing SMA medicine (risdiplam) can help people with spinal muscular atrophy build stronger muscles and move better. The trial includes about 259 children and young adults, ages 2 to 25, who can walk. Resear…

This study looks at whether the drug troriluzole can slow the progression of spinocerebellar ataxia (SCA), a rare genetic disease that affects movement and balance. Researchers will compare 909 patients who took troriluzole for up to three years with similar patients who did not …

This Phase 3 trial tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy. Participants receive either the cell therapy or a placebo every 3 months for a year, then all can receive the therapy for another year. The goal is to …

This study tests whether the drug tafamidis can safely stop the progression of ATTR amyloidosis in people who have already received a heart transplant. About 25 stable patients will take tafamidis daily for 12 months. Researchers will measure changes in blood protein levels, nerv…

This Phase 2 trial tests two experimental drug combinations in 63 women with a specific type of ovarian cancer (BRCA-mutant or HRD-positive) that has worsened after prior PARP inhibitor treatment. Participants receive either tuvusertib plus niraparib or tuvusertib plus larteserti…

This study tests a single dose of SGT-001 gene therapy in 12 boys (children and teens) with Duchenne muscular dystrophy. The main goal is to check safety and how well the body tolerates the treatment. Participants will be followed for about 5 years to monitor side effects and any…

This study tests a one-time gene therapy infusion (FBX-101) given after a standard stem cell transplant in 6 children with infantile Krabbe disease, a severe genetic disorder affecting the nervous system. The therapy uses a harmless virus to deliver a working copy of the GALC gen…

This early study tests a single injection of a gene therapy called NTF001 in 12 people with ALS. The therapy aims to deliver a protein that supports nerve cell health. The main goal is to check safety, but researchers will also look for signs of benefit over 52 weeks.

This study looks at whether giving people with PTSD a personalized mix of gut bacteria (called pharmabiotics) can help their mental health. The researchers will track changes in PTSD symptoms, anxiety, depression, and thinking skills over 14 days. The goal is to see if fixing the…

This study tests whether adding taldefgrobep alfa to standard SMA treatments can improve muscle function in people with spinal muscular atrophy (SMA). About 269 participants, both walkers and non-walkers, will receive either the drug or a placebo for 48 weeks. The main goal is to…

This early-stage trial tests a new gene therapy called NKG001 in 21 children under 5 with spinal muscular atrophy (SMA). The therapy is given as a single dose, either through a vein or combined with a spinal injection. The main goal is to check safety and find the best dose, not …

This early-stage study tests the safety of acamprosate, a drug used for alcohol dependence, in 30 adults with ALS who have a specific mutation in the C9orf72 gene. Participants take the pill three times daily for 24 weeks and undergo regular checkups, breathing tests, and spinal …

This Phase 3 trial tests remibrutinib, an oral tablet, against teriflunomide in 1000 adults with relapsing multiple sclerosis. The goal is to see if remibrutinib better reduces yearly relapse rates and delays disability. Participants will then have the option to continue remibrut…

This study was designed to test whether the drug govorestat could improve symptoms of Charcot-Marie-Tooth disease caused by sorbitol dehydrogenase deficiency (CMT-SORD), a rare nerve condition. It planned to enroll people aged 16 to 65 and compare govorestat to a placebo over 36 …

This study tests a new drug, Tuvusertib, combined with an immunotherapy called Cemiplimab in people with a specific type of advanced lung cancer (non-squamous non-small cell) that has worsened after prior treatments. The trial aims to see if the combination can shrink tumors and …

This Phase 3 trial tests a gene therapy called SRP-9001 for Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. It includes 148 males who can and cannot walk. Participants receive a one-time IV infusion of the therapy or a placebo, and are followed for about 128 w…

This pilot study aimed to test whether a mixture of dexamethasone, hyaluronic acid, and a green dye could be safely injected into the ear to treat certain types of hearing loss that don't improve with standard treatments. The plan was to enroll 26 adults with sudden hearing loss,…

This study tests a higher dose of nusinersen (Spinraza) in 45 teenagers and adults with spinal muscular atrophy (SMA) who cannot walk and have already taken risdiplam. Participants receive two 50 mg loading doses two weeks apart, then 28 mg every four months for about two years, …

This study tests whether adding telitacicept to standard therapy helps adults with refractory chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve condition that causes weakness and numbness. About 76 participants will receive either standard care alone or standard c…

This phase 2 trial tests a daily oral drug called sevasemten (EDG-5506) in 244 adults and adolescents with Becker muscular dystrophy, a condition that causes progressive muscle weakness. The study compares the drug to a placebo to see if it can reduce muscle damage, improve walki…

This study tests if a new drug, empasiprubart, works as well as standard IVIg treatment for adults with multifocal motor neuropathy (MMN), a condition that causes muscle weakness. About 154 people who have responded to IVIg in the past will be randomly assigned to receive either …

This study tests satralizumab, a drug that calms inflammation, in 30 children aged 8 to 17 with Duchenne muscular dystrophy. The goal is to see if it improves bone density and muscle function. Participants receive injections for several months while continuing standard steroid th…

This study tests whether a cochlear implant can safely and effectively restore hearing in people who become deaf in one ear after surgery to remove a benign tumor (vestibular schwannoma) or to treat Meniere's disease. Fifteen participants will receive the implant during their sur…

This early-stage trial tests a gene therapy called PBFT02 for frontotemporal dementia (FTD) in people with specific genetic mutations. The therapy delivers a working copy of the GRN gene directly into the brain. The study involves 30 adults and primarily checks for safety and sid…

This study is for people with Duchenne muscular dystrophy (DMD) who have already taken part in a previous trial of BMN 351. It tests the long-term safety of weekly intravenous infusions of BMN 351 and whether it helps maintain physical function. Only 18 participants will be enrol…

This early-stage study tests whether a new drug called QRL-201 is safe and tolerable for people with ALS (Lou Gehrig's disease). About 69 adults aged 18-80 with recent ALS symptoms will receive either the drug or a placebo. The main goal is to check for side effects, not to cure …

This study is for people with FSHD, a genetic disease that causes muscle weakness. It tests the long-term safety and how well the body tolerates a drug called AOC 1020, given through a vein. About 84 adults who completed a previous study will take part. The main goal is to check …

This study tests a gene therapy called delandistrogene moxeparvovec in 13 children under age 4 with Duchenne muscular dystrophy. The goal is to see if it is safe and can help produce a key muscle protein. The children will be followed for about 5 years.

This study was designed to test whether a daily handgrip exercise program could speed up nerve regrowth and restore muscle and bone after brachial plexus injury surgery. It planned to enroll adults aged 18–60 with confirmed nerve damage. However, the trial was withdrawn before an…

This phase 2 trial tests whether the drug zanubrutinib, combined with standard rituximab, can improve nerve damage and disability in people with a rare blood condition called IgM MGUS. The study includes 35 adults across two Dutch hospitals. Participants take zanubrutinib daily f…

This was a planned phase 2 trial to test IC14, a monoclonal antibody, in 50 people with rapidly progressing ALS. Participants would have received intravenous IC14 or placebo twice weekly for 12 weeks. The study was withdrawn before any patients were enrolled, so no data on safety…

This study planned to test an experimental drug called IC14 in people with rapidly progressing ALS. The drug was given by IV over two weeks, and brain scans measured inflammation. However, the trial was withdrawn before enrolling any participants, so no data on safety or effectiv…

This study looks at the long-term safety and effects of a drug called EDG-5506 (sevasemten) in people with Becker muscular dystrophy, a muscle-weakening disease. About 260 adults and teens who were in earlier EDG-5506 studies will take the drug and be monitored for side effects a…

This study follows 500 people with Duchenne muscular dystrophy to see how well a gene therapy (ELEVIDYS) works compared to standard steroid treatment over time. Researchers will measure movement abilities, breathing, and safety, including liver problems. Participants must already…

This expanded access program provides apitegromab, an experimental drug that blocks myostatin to help maintain muscle strength, to eligible patients with spinal muscular atrophy (SMA) before it is officially approved. Participants must be at least 2 years old and have a confirmed…

This early-stage study tests whether a new drug, prosetin, is safe for people with ALS (Lou Gehrig's disease) and healthy volunteers. About 72 participants will receive either the drug or a placebo to check for side effects and how the drug moves through the body. The goal is to …

This pilot study tests a device called Travagus One, which stimulates a nerve in the ear to reduce inflammation in boys with Duchenne muscular dystrophy (DMD). Twenty boys aged 5-17 will use the headset at home for 5 minutes twice a day for one week. Researchers will measure infl…

This study follows about 80 boys with Duchenne muscular dystrophy who have already taken vamorolone in earlier studies. Researchers want to see how safe the drug is over a longer time, especially its effect on bone health, like spine fractures. The boys continue taking vamorolone…

This study tests the drug alpelisib, already approved for some breast cancers, in people aged 2 months to 30 years with certain blood vessel growth disorders caused by PIK3CA or TIE2 gene changes. Participants take the drug for 2-3 years and attend up to 16 clinic visits to check…

This study looks at the long-term safety and effects of a drug called SAT-3247 in 10 people with Duchenne muscular dystrophy who were in a previous trial. Participants take the drug by mouth five days a week for about 11 months. Researchers will check for side effects and measure…

This phase 3 study is testing the long-term safety and effectiveness of an experimental drug called AOC 1001 (del-desiran) for people with myotonic dystrophy type 1 (DM1), a genetic muscle disorder. The trial enrolls 230 adults who have completed a prior AOC 1001 study and will r…

This early trial tests whether a transplant of umbilical cord blood can help people with ALS, a fast-progressing nerve disease. Eight adults will receive the transplant after a mild chemotherapy-like prep. Researchers will check safety and whether the treatment slows decline in m…

This study follows 403 adults and children with spinal muscular atrophy who are already taking risdiplam (Evrysdi) as prescribed by their doctor. Researchers will track side effects and how well the drug works over about two years. The goal is to see if risdiplam remains safe and…

This study tests a drug called SHP611, given as a spinal injection, in 36 children with late infantile metachromatic leukodystrophy (MLD), a rare and severe brain disease. The main goal is to see if the treatment helps children keep their ability to walk and move for as long as p…

This study aimed to see if a mist-inhaled drug called RNS60 could help people with ALS, a nerve disease that weakens muscles. It planned to measure changes in daily function, breathing, and survival. However, the trial was withdrawn before enrolling any participants, so no result…

This Phase 2 trial is testing a new drug called NVG-2089 in 60 adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a condition where the immune system attacks nerves. The drug is designed to mimic a natural protein (IVIg) to reduce inflammation and improve nerve…

This program offered vatiquinone, an experimental liquid medication, to patients with inherited mitochondrial diseases like Leigh syndrome who had already completed a previous safety study. The goal was to continue treatment for those who might benefit, but enrollment is now clos…

This study tested an experimental drug called ARGX-119 in 60 adults with ALS. The goal was to see if it was safe and could help with muscle function. The trial was stopped early because early results showed it was unlikely to make a meaningful difference. No safety concerns trigg…

This early-phase trial tests the safety and tolerability of a new drug called GRT6019 in 24 healthy men. Participants receive multiple doses over 4 weeks and are monitored for side effects and how the drug moves through the body. The goal is to see if GRT6019 is safe enough to st…

This study tests the drug risdiplam (Evrysdi) in infants up to 6 weeks old who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The goal is to see if early treatment can help them reach motor milestones like sitting without support. The trial involve…

This program provides early access to an experimental drug called CNM-Au8 for people with amyotrophic lateral sclerosis (ALS), a progressive nerve disease. Participants must be 18 or older and have a confirmed ALS diagnosis. The goal is to offer treatment to those who cannot join…

This program provides CNM-Au8, a suspension of gold nanocrystals, to up to 300 people with ALS. The main goal is to give access to the drug and monitor safety, not to test if it works. Participants must have a confirmed ALS diagnosis and be at least 18 years old.

This early study tests whether fish oil, alone or with salsalate (an anti-inflammatory drug), can improve blood markers linked to nerve health in people with type 2 diabetes and diabetic neuropathy. About 100 adults will take fish oil capsules for 4 months, then add salsalate for…

This early-stage trial tests a daily injection of ASA-001 in two adults with a rare genetic muscle disease (ADSS1 deficient myopathy). The main goals are to see if the drug is safe and if it can slow the disease. Participants will take the drug for 8 months and have clinic visits…

This study tests a new drug called 9-ING-41, given alone or with chemotherapy, in adults with cancers that have not responded to standard treatments. The goal is to see if the drug is safe and can help control the disease. About 350 people with various advanced cancers, including…

This phase 3 trial tests whether the drug tofersen can delay or prevent ALS in adults who carry a SOD1 gene mutation but have no symptoms yet. About 158 participants will receive either tofersen or a placebo, and researchers will track how many develop ALS within two years. The g…

This study tests the drug sirolimus (rapamycin) in 15 people aged 6 months to 55 years with genetically confirmed Leigh syndrome, a rare and serious mitochondrial disease. The goal is to see if the drug is safe and can help with symptoms like muscle weakness and developmental del…

This study follows 66 boys with Duchenne muscular dystrophy who previously received RGX-202 gene therapy. Researchers will monitor side effects and measure muscle function over time, such as how fast they can stand, walk, or climb. The goal is to see if the treatment remains safe…

This study tests an oral drug called emavusertib (CA-4948) in adults with acute myeloid leukemia (AML) or a high-risk bone marrow disorder (MDS) that have not responded to or returned after prior treatment. The goal is to find the safest and most effective dose and to see if the …

This study tests a new painless nerve growth factor eye drop (CHF6467) in 36 children and young adults with optic pathway glioma, a brain tumor that often causes vision loss. The goal is to see if the drops can protect or improve vision without the pain side effects of standard n…

This early-stage trial tests a single dose of a gene therapy called JWK007 in 3 boys aged 5-10 with Duchenne muscular dystrophy. The main goal is to check safety and tolerability, while also looking at whether it helps muscle function. It is a very small, first-in-human study, so…

This pilot study tests whether combining intensive hand-arm and leg therapy with strength training can improve motor function in children with spinal muscular atrophy (SMA). Five children will attend one 6-hour session each weekend for 15 weeks. Researchers will check if the ther…

This early-phase study is testing whether stem cell therapies and experimental drugs can help repair inner ear damage in people with hearing loss or tinnitus. Researchers will monitor 120 adults over time using hearing tests and symptom surveys. The goal is to see if these regene…

This study is a long-term follow-up for people with multifocal motor neuropathy, a rare condition that causes muscle weakness. It tests the safety and effectiveness of a drug called ARGX-117, given intravenously. The trial includes 51 adults who previously took part in an earlier…

This study is testing a new device called a multichannel vestibular implant for people who have lost most or all of their balance function due to inner ear damage. The implant electrically stimulates the balance nerve to help improve stability and vision during movement. Up to 30…

This study tests a new treatment for ALS (Lou Gehrig's disease) using stem cells taken from the patient's own belly fat. The cells are injected into the fluid around the spine every 3 months for a year. The goal is to see if this treatment is safe and can slow down the disease. A…

This clinical trial tests whether ibudilast (MN-166), a drug previously used for asthma, can slow the progression of amyotrophic lateral sclerosis (ALS). The study involves 234 adults with ALS, who will receive either ibudilast or a placebo for 12 months, followed by a 6-month op…

This study looks at the long-term safety of an experimental drug called WVE-N531 in people with Duchenne muscular dystrophy (DMD) who have already taken it in a previous study. About 175 participants will receive the drug and be monitored for side effects, heart and lung function…

This study tests a single dose of SRP-9003 gene therapy in 17 people with limb girdle muscular dystrophy 2E/R4, a genetic muscle-weakening disease. The goal is to restore a missing protein in muscle cells and improve muscle function. Both walkers and non-walkers can join, and the…

This study tests a gene therapy called PF-06939926 in 114 boys with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. Two-thirds receive the gene therapy, while one-third get a placebo, but can switch to the real treatment after one year. The main goal is to see…

This study tests a surgical procedure that moves two tendons in the forearm to help people with long-term ulnar nerve damage regain the ability to pinch. 17 adults aged 18-60 with stable joints and no other nerve injuries will be followed for 12 weeks after surgery. The goal is t…

This Phase 3 trial tests whether higher doses of eteplirsen (Exondys 51) can safely improve muscle function in boys with Duchenne muscular dystrophy whose genetic mutation allows exon 51 skipping. About 160 ambulatory boys will receive one of three doses (30, 100, or 200 mg/kg) t…

This phase 2 trial tests whether high-dose chemotherapy followed by a stem cell transplant can stop the immune system from attacking the nervous system in people with autoimmune neurologic diseases like multiple sclerosis and stiff person syndrome. The treatment aims to weaken th…

This study is for people with a rare heart condition called ATTR amyloidosis with cardiomyopathy, where abnormal protein builds up in the heart. It tests the long-term safety and effectiveness of a drug called vutrisiran, given as an injection every three months. About 700 adults…

This early-phase trial tests a single-dose gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can produce the missing beta-sarcoglycan protein …

This study tests an experimental drug called ION363 in people with a rare, inherited form of ALS caused by FUS gene mutations. The goal is to see if the drug can slow the disease and help people live longer. About 89 participants will receive the drug via spinal injection. This i…

This study tests a stem cell transplant using a milder chemotherapy-like regimen to treat children and young adults (up to age 28) with various immune system disorders. The goal is to help the donor stem cells settle in the body with fewer side effects. The study involves 20 part…

This long-term study is testing higher doses of nusinersen (Spinraza) in people with spinal muscular atrophy who already completed a previous nusinersen study. The main goal is to check safety over several years, including side effects, heart health, and growth. Participants rece…

This clinical trial is testing an oral drug called NUZ-001 in 160 people with ALS. Participants take the drug or a placebo daily for 36 weeks. The study measures how the disease progresses using a scale of physical function and survival. The goal is to see if NUZ-001 can slow ALS…

This study tests a surgery called lymphovenous bypass for people with diabetic peripheral neuropathy, a common complication causing pain, numbness, and foot ulcers. The procedure aims to drain excess lymph fluid, which may reduce inflammation and nerve damage. Researchers will me…

This trial provides expanded access to RAPA-501, a therapy made from a patient's own immune cells, for people with ALS. The goal is to control the disease and slow its progression. Participants must be 18 or older and have a confirmed ALS diagnosis.

This study tests whether breathing short bursts of air with slightly less oxygen can help nerves in the hand heal better after injury. The therapy is non-invasive and aims to improve hand function and reduce pain. About 80 adults with severe carpal tunnel syndrome will be enrolle…

This study tests whether the drug usnoflast can slow the progression of ALS, a severe nerve disease that causes muscle weakness and breathing problems. About 240 adults with ALS will receive either usnoflast or a placebo for 36 weeks. Researchers will measure changes in daily fun…

This early-stage trial tests a single intravenous dose of a gene therapy (ATA-200) in 4 children aged 6-12 with limb-girdle muscular dystrophy type 2C/R5 (LGMD2C), a rare genetic muscle-weakening condition. The goal is to see if the treatment is safe and tolerable by delivering a…

This study follows over 3,000 people who received the Avance Nerve Graft to repair damaged peripheral nerves. Researchers are tracking safety, sensory and motor function recovery, and quality of life in real-world settings. The goal is to understand how well this processed human …

This phase 3 trial tests an experimental drug called AOC 1001 in 159 adults with myotonic dystrophy type 1, a genetic condition that causes muscle weakness and stiffness. Participants receive either the drug or a placebo by intravenous infusion. The main goal is to see if the dru…

This early-phase trial tests a new drug called RAG-18 in 12 boys aged 4-15 with Duchenne muscular dystrophy. The drug is a small activating RNA given through an IV, designed to help muscles work better. The main goal is to check safety and how the body handles the drug, with earl…

This study tests whether a combination of nicotinamide (a form of vitamin B3) and pterostilbene (a plant compound) can slow the progression of ALS, a fast-moving nerve disease. About 380 adults with ALS will take either the supplement or a placebo for a period of time. The goal i…

This study tests a drug called vutrisiran in 655 adults with a rare heart condition caused by abnormal protein buildup (ATTR amyloidosis with cardiomyopathy). The drug is given as a shot every 3 months and aims to reduce deaths and heart-related hospital stays. The goal is to see…

This study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to get the donor cells to take hold while keeping side effects low. Participants receive chemotherapy drugs before the transplant to prepare their bo…

This study checks the long-term safety of a drug called batoclimab for adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder that causes weakness and numbness. About 108 people who finished an earlier batoclimab study will get weekly injections fo…

This study tests a new drug called NX210c in 80 adults with ALS, a nerve disease that weakens muscles over time. Participants receive multiple IV infusions of the drug or a placebo to see if it slows the disease and is safe. The main focus is on changes in blood markers that refl…

This study is an open-label extension for people with early amyotrophic lateral sclerosis (ALS) who completed a previous placebo-controlled trial. Participants will take a daily oral dose of CNM-Au8, a suspension of gold nanocrystals, to see if it safely slows disease progression…

This early-stage trial tests an experimental oral drug called PRJ1-3024 in 39 adults with advanced solid tumors that have not responded to standard treatments. The main goal is to check safety and find the right dose. Researchers will also look for early signs that the drug may s…

This study tests whether combining acalabrutinib with rituximab is safe and effective for people with nerve damage (neuropathy) linked to two blood conditions: IgM MGUS or Waldenstrom macroglobulinemia. The main goal is to see if the treatment lowers a key protein (IgM) in the bl…

This study tests whether wrapping the spinal accessory nerve with a porcine (pig) tissue product during neck dissection for head and neck cancer can prevent shoulder problems. About 74 adults who have not had prior neck surgery will be enrolled. Researchers will measure shoulder …

This study looks at the long-term safety and effects of a drug called eplontersen in people with a rare inherited nerve disease called hATTR-PN. About 151 participants who completed earlier studies will receive regular injections of eplontersen. The goal is to see if the drug rem…

This study tests whether metformin, a widely used diabetes drug, is safe for people with a specific genetic form of ALS (C9orf72). Over 24 weeks, 41 participants will take metformin to see if it reduces toxic proteins linked to the disease. The goal is to determine if metformin c…

This study follows 20 adults with spinal muscular atrophy (SMA) type II who are already taking SPINRAZA. Researchers will measure motor function using a 32-point scale over 27 months to see if the drug helps maintain or improve movement. The goal is to better understand how SPINR…

This study planned to test a gene therapy called VM202 in people with painful diabetic neuropathy who were already taking opioid painkillers. The goal was to see if VM202 could help them safely reduce their opioid dose. However, the trial was withdrawn before any participants wer…

This early-phase trial tests a single infusion of a gene therapy called FBX-101 in 9 people with Krabbe disease who have already received a stem cell transplant. The therapy uses a harmless virus to deliver a working copy of the GALC gene, aiming to improve motor function and saf…

This early-stage trial tests a single injection of RJK002, a gene therapy, into the spinal fluid of 9 people with ALS. The main goal is to see if it is safe and to find the best dose for future studies. Researchers will also track changes in daily function, but the trial is too s…

This study tests a new gene therapy called SKG0201 in 12 infants with spinal muscular atrophy type 1, a severe muscle-weakening disease. The treatment aims to improve survival and motor skills by delivering a working gene. Researchers are checking safety and how well the therapy …

This study looks at people with relapsing-remitting multiple sclerosis who switched from the drug MabThera to a similar, lower-cost medicine called Rixathon. Researchers want to see if the switch changes signs of nerve damage, new brain lesions, relapses, or disability. They will…

This study is testing whether low-dose lithium aspartate, a dietary supplement, can slow the progression of Parkinson's disease. It involves 35 people who have already completed a lithium trial, and they will take lithium for another 24 weeks. Researchers will use brain scans and…

This study tests an experimental drug called sevasemten in 76 children aged 4-9 with Duchenne muscular dystrophy. The goal is to check the drug's safety, how the body processes it, and its effect on muscle health markers. Participants are randomly assigned to receive the drug or …

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with Charcot-Marie-Tooth disease type 2D (CMT2D) caused by a specific GARS1 gene mutation. The drug aims to improve motor skills and quality of life. Only one participant is enro…

This study tests an experimental drug called EDG-5506 (sevasemten) in 43 children and teens aged 6 to 17 with Duchenne muscular dystrophy who have already received gene therapy. The goal is to see if the drug is safe and how it affects muscle damage markers. Participants are rand…

This study tests the long-term safety and effectiveness of a cell therapy called deramiocel (CAP-1002) in people with Duchenne muscular dystrophy who completed the earlier HOPE-2 trial. Participants receive an infusion of deramiocel every 3 months for about 5 years, with the opti…

This study tests a custom-made drug (VCA-894A) in a single person with a rare genetic nerve disease called CMT2S. The drug is designed to correct a specific genetic error and restore a missing protein. The main goals are to check if the treatment is safe and if it can improve mus…

This study tests whether digoxin, a heart medication, is safe and tolerable for people with ALS. It also looks at whether digoxin can slow nerve damage. About 40 adults with early-stage ALS will take digoxin for 24 weeks. The goal is to find a new way to manage this disease.

This early-stage trial tests a gene therapy called AMT-162 in 20 adults with a specific genetic form of ALS (SOD1-ALS). The treatment is given as a single injection into the spinal fluid. The main goal is to check safety and tolerability, while also looking for early signs that i…

This study follows 7 people who previously received a gene therapy called AXO-AAV-GM2 for Tay-Sachs or Sandhoff disease — rare, fatal genetic disorders that destroy nerve cells. Researchers will track them for up to 5 years to see if the treatment remains safe and whether it help…

This early-stage trial tests a gene therapy called ATA-100 for people with LGMDR9, a rare genetic muscle disease that causes progressive weakness. Six adults receive a single intravenous infusion of the therapy, which delivers a working copy of the FKRP gene. The main goal is to …

This study aimed to test if daratumumab could improve nerve damage (peripheral neuropathy) in people with a blood condition called MGUS. Participants would have received weekly injections for two months, then biweekly for four more months. However, the trial was withdrawn before …

This early-stage trial tests a single injection of gene therapy for people with SMARD1 or CMT2S, rare diseases caused by mutations in the IGHMBP2 gene. The therapy delivers a working copy of the gene directly into the spinal fluid. Ten participants, ranging from infants to childr…

This study gives one patient with CIDP access to their own stored stem cells to see if it's safe and might help. The patient receives multiple infusions over 44 weeks and is followed for a year. Because it's only one person, the results are very limited.

This trial gives a single child with a rare form of congenital muscular dystrophy access to their own banked stem cells. The cells are given through 14 IV infusions to see if they are safe and can help control the disease. Because it involves only one patient, the results will be…

This study provides an experimental stem cell treatment (HB-adMSCs) to an 83-year-old man with Primary Lateral Sclerosis, a rare nerve disease that causes progressive muscle weakness. The patient's own banked stem cells are used, aiming to slow the disease. This is a single-patie…

This study tested a stem cell treatment for amyotrophic lateral sclerosis (ALS), a progressive nerve disease. It involved one patient who received their own fat-derived stem cells to try to control the disease. The treatment is no longer available.

This study tests a drug called BBP-418 (Ribitol) in 81 people aged 12 to 60 with limb girdle muscular dystrophy type 2I (LGMD2I), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo for 36 months to see if it slows the di…

This study tests a drug called pacritinib in people with advanced solid tumors (like breast cancer) that have a specific genetic change called 1q21.3 amplification. The goal is to see if the drug can shrink tumors or slow their growth. About 74 adults whose cancer has not respond…

This study tests two drugs, nivolumab and cabozantinib, together in 60 people with advanced or metastatic non-clear cell kidney cancer. The goal is to see how well the combination shrinks tumors. Participants take cabozantinib daily and receive nivolumab infusions every two weeks…

This study tests the long-term safety and effectiveness of an experimental drug called DS-5141b (Renadirsen) in 8 people with Duchenne muscular dystrophy who have already completed a previous study. Participants receive a weekly injection under the skin. Researchers will monitor …

This study planned to test the drug rituximab in people with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve condition that causes weakness and numbness. The goal was to see if rituximab could help certain patients stop their current treatments and achieve long-…

This early-phase trial tests a substance made from stem cells from baby teeth (SHED-CM) in 10 people with ALS. The goal is to see if it is safe and if it might slow the disease. Participants receive the treatment intravenously over 12 weeks, with follow-up for 4 more weeks.

This Phase 3 trial tests whether TAS-205, an oral drug, can improve movement and safety in people with Duchenne muscular dystrophy. It includes 104 participants, both those who can walk and those who cannot. The study compares TAS-205 to a placebo over 52 weeks.

This UK trial tests whether cladribine tablets, taken for just 8-10 days a year over two years, can slow the loss of hand function in people with advanced multiple sclerosis (MS). The study includes 204 participants with significant disability (EDSS 6.5-8.5). Half receive cladrib…

This study tests a single infusion of OTL-200 gene therapy in 6 people with late juvenile metachromatic leukodystrophy (MLD), a rare genetic disease that damages the brain and nerves. The therapy uses the patient's own blood stem cells, modified to produce a missing enzyme. Resea…

This program gives ALS patients who cannot join other clinical trials access to an experimental drug called trehalose (SLS-005). Trehalose is a sugar solution given once a week through a vein. The goal is to see if it can help control the disease. The program is no longer availab…

This early study will test whether weak electrical currents applied to the head can help diagnose balance disorders like vestibular schwannoma and vestibular neuritis. Researchers will measure how 30 people with these conditions sway or move in response to the stimulation. The go…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study compares a new ultra-high resolution PET/CT scanner, the NeuroEXPLORER, with standard clinical PET/CT scanners for imaging the head and neck. Researchers aim to see if the new device provides more detailed images to improve diagnosis of conditions like dementia, Parkin…

This study is testing whether a simple saliva sample can help diagnose amyotrophic lateral sclerosis (ALS). Researchers will analyze RNA in the saliva of 653 people, including ALS patients and healthy volunteers. If the test works, it could offer a faster, less invasive way to di…

This study explores whether ultrasound elastography, a non-invasive imaging technique, can accurately diagnose carpal tunnel syndrome (CTS) without the need for painful needle-based nerve tests. Researchers will enroll 80 participants—some with CTS and some healthy volunteers—to …

This study looks at whether a special brain scan called diffusion tensor imaging (DTI) can help doctors find Krabbe disease in newborns and decide who needs urgent treatment. About 100 babies with a positive newborn screen or family history of Krabbe disease will be scanned and f…

This study is testing a way to find cardiac amyloidosis—a rare but serious heart condition—earlier in people aged 65 and older. Researchers in Turkey will screen 800 patients at family medicine clinics using simple blood tests and medical history. The goal is to see if this appro…

This study aimed to test a computer algorithm that uses insurance claims data to identify people with ATTR amyloidosis, a rare disease that affects the heart and nerves. Researchers planned to check how well the algorithm worked by reviewing medical records. However, the study wa…

This study aims to create a simple test battery to determine fall risk in people with neuromuscular disorders, such as muscular dystrophy or ALS. Researchers will assess 108 participants using several physical tests like walking, standing, and rising from a chair. The goal is to …

ScreenPlus is a large pilot program that offers families the option to have their newborn screened for a panel of rare genetic disorders, in addition to standard newborn screening. The study aims to screen 100,000 infants born at eight hospitals in New York. Researchers will eval…

This study tests a short questionnaire called ACT to see if it can accurately detect nerve damage in the feet and legs of people with diabetes. Researchers will compare it to standard screening tools and an eye test that looks at tiny nerve fibers. The goal is to find a simple, n…

This trial tests a wearable insole system that monitors foot pressure, temperature, and movement, giving real-time alerts to help users shift weight and avoid injury. The study includes people with type 1 or 2 diabetes who have nerve damage and a recent history of healed foot ulc…

This study was designed to test whether an oral drug called SPI-1005 could prevent hearing loss and ringing in the ears caused by platinum-based chemotherapy. It was planned for adults with advanced lung or head and neck cancer. The study was withdrawn before enrolling any partic…

This study tests a natural substance called GM1 to see if it can prevent nerve damage (pain, numbness, tingling) caused by the chemotherapy drug paclitaxel in people with metastatic breast cancer. About 98 participants will receive either GM1 or a placebo alongside their chemo. T…

This phase II trial tests whether riluzole, a drug used for ALS, can prevent nerve damage caused by oxaliplatin chemotherapy in people with stage II/III colorectal cancer. About 80 participants will receive either riluzole or a placebo during their chemo. The study is currently s…

This study tests whether wearing cold gloves during chemotherapy can prevent peripheral neuropathy (nerve damage causing numbness, tingling, or pain) in colon cancer patients. Forty adults with stage III colon cancer who are about to start a common chemo regimen (CAPOX) will be r…

This study tests whether wearing two layers of surgical gloves during paclitaxel chemotherapy can prevent nerve damage in the hands. About 104 breast cancer patients will be randomly assigned to wear the gloves or not. Researchers will compare changes in nerve pain and function b…

This phase II/III trial tests whether duloxetine, a drug used for depression and pain, can prevent the nerve damage (pain, tingling, numbness) caused by oxaliplatin chemotherapy in stage II-III colorectal cancer patients. About 220 participants will receive either duloxetine or a…

This study tests whether cookies made from golden rice and red betel leaf can help prevent diabetic neuropathy, a common nerve damage complication of diabetes. 102 adults with diabetes will eat one cookie daily for 28 days. Researchers will measure changes in nerve symptoms and b…

This study looks at whether foot and ankle exercises using elastic bands can help prevent diabetic foot ulcers in people with diabetic neuropathy. About 80 participants with type 2 diabetes and nerve damage in their feet will do these exercises. Researchers will track how many ul…

This study tests a small implanted device that sends mild electrical pulses to nerves to relieve chronic pain from injury or surgery. About 89 adults with long-lasting pain in the back, shoulder, knee, or foot will either get the implant plus usual care or usual care alone. The g…

This study tests whether a device that sends mild electrical pulses to the spinal cord (10 kHz SCS) can reduce pain and improve nerve function in people with painful diabetic neuropathy. About 143 participants will either get standard medical care alone or the device plus standar…

This study tests whether two non-invasive brain stimulation techniques—transcranial Direct Current Stimulation (tDCS) and Transcranial Ultrasound (TUS)—can reduce chronic pain in people with diabetic neuropathy. Participants, aged 40 to 80 with foot pain lasting at least 6 months…

This study looks at how well a spinal cord stimulator works in real-world settings for people with long-term leg pain caused by diabetic neuropathy. About 500 participants will receive the implant and be followed for a year. Researchers will measure pain relief, quality of life, …

This phase 2 trial tests whether combining neurofeedback (a brain-wave training therapy) with the drug duloxetine can better relieve nerve pain caused by chemotherapy than either treatment alone. About 336 cancer survivors with persistent nerve pain will be randomly assigned to o…

This study tests a non-invasive light therapy called photobiomodulation (low-level laser) for people with diabetic peripheral neuropathy—nerve damage causing pain, tingling, and numbness in the feet. 36 adults with type 2 diabetes and foot pain for at least 6 months will receive …

This study tests whether two devices, used one after the other, can reduce pain and disability from temporomandibular joint (TMJ) disorders. Adults aged 22 and older with TMJ pain will receive four treatment sessions and be followed for three months. Researchers will measure chan…

This study tests whether photobiomodulation (low-level laser therapy) can reduce pain and disability in people with anti-MAG neuropathy, a rare nerve condition. Ten participants will receive 16 laser sessions over six weeks. Researchers will measure changes in disability, pain, a…

This small early-phase trial tests whether a single oral dose of a hemp-derived CBD/CBD-A extract can reduce neuropathic pain and change brain activity in people with spinal cord injury. Six participants with moderate to severe nerve pain for at least three months will receive ei…

This study tests a procedure called cryoneurolysis, which uses a small probe to freeze nerves in the foot and block pain signals. It is for adults with painful diabetic neuropathy who have at least moderate pain daily. Thirty participants will be randomly assigned to receive eith…

This study tests whether 3D printed custom insoles are better than regular ready-to-wear insoles at reducing foot pressure in people with diabetic neuropathy. High foot pressure can lead to ulcers and mobility problems. Twenty-two adults with diabetes and nerve damage will be ran…

This study investigates whether foot massage using a roller can reduce nerve pain caused by chemotherapy in cancer patients. About 80 adults receiving certain chemotherapy drugs will either receive foot massage twice daily for three weeks or continue with routine care. The goal i…

This pilot study tests whether a vibrating device strapped to the leg can improve walking in young cancer survivors (ages 6-39) who have nerve damage from chemotherapy. Ten participants will have the device applied to leg muscles while their step times are measured. The goal is t…

This study tests a personalized workplace intervention for 124 Dutch workers with Parkinson's disease, cerebellar ataxia, hereditary spastic paraparesis, or slowly progressive neuromuscular/mitochondrial disorders. A trained facilitator helps employees and their managers identify…

This study tests two types of exercises to help people who get dizzy, nauseous, or have blurry vision from using computers for long hours. 46 computer users will do either head-movement or eye-movement exercises for 4 weeks. The goal is to see which exercise better reduces sympto…

This study tests whether a pain-relief gel (pregabalin) delivered through the skin using ultrasound can reduce pain and improve hand function in people with carpal tunnel syndrome. The treatment is given four times a week for a month, and compared to standard ultrasound therapy w…

This study tests whether giving Botox injections early can reduce nerve pain that occurs after chest surgery (thoracoscopy or thoracotomy). Participants have had daily nerve pain for three months. The trial compares Botox to a placebo to see if it lowers pain intensity and preven…

This study tests whether a 6-week, caregiver-assisted breathing program done at home with remote coaching is practical and helpful for children with Duchenne muscular dystrophy. Twelve children will do breathing exercises, airway clearance, and relaxation techniques at least 5 ti…

This study tests whether a 10-week, at-home exercise program is safe and helpful for cancer survivors who have ongoing nerve pain, numbness, or tingling from chemotherapy. About 40 participants will do remote exercise sessions and health coaching calls. The goal is to see if the …

This study tests two different exercise programs in 40 people with diabetic peripheral neuropathy, a condition that causes numbness and balance problems. One program uses Feldenkrais exercises, which focus on movement awareness, and the other uses task-oriented gait training, whi…

This study tests a special nerve cap (Axoguard Large-Diameter Nerve Cap) in 15 adults who have had a limb injury or amputation. The cap is placed over the cut nerve end to protect it and may help reduce chronic nerve pain. The goal is to see if the cap is safe and works well befo…

This study tests a treatment called neuromodulation to see if it can reduce nerve pain caused by chemotherapy. About 24 adults with nerve pain in their legs from certain chemo drugs will take part. Researchers will check for side effects and measure changes in pain and nerve func…

This study looks at whether Scrambler therapy can safely reduce painful nerve damage caused by chemotherapy in adolescents and young adults (ages 15-39) who have finished active cancer treatment. Participants are randomly assigned to receive the therapy right away or after a wait…

This small pilot study tests whether a closed loop spinal cord stimulator can reduce pain and improve quality of life for people with nerve damage from chemotherapy. Ten adults with chemotherapy-induced peripheral neuropathy in their legs will receive the device and be monitored …

This study tests a drug called suzetrigine (VX-548) for long-term safety and pain relief in people with diabetic peripheral neuropathy. About 455 adults who completed a prior 12-week study will take the drug for up to one year. Researchers will track side effects and changes in p…

This study looks at whether a non-surgical treatment called spinal decompression can help people with chronic low back pain. About 42 adults with back pain from disc problems or sciatica will receive the treatment over 12 weeks. Researchers will use MRI scans and movement tests t…

This phase 2 trial tests a special hand and foot mask designed to ease nerve pain caused by chemotherapy or diabetes. Sixty adults will apply the mask daily for 5 minutes over one week. Researchers will measure changes in symptoms and quality of life using questionnaires.

This study looks at whether taking vitamin D can reduce nerve pain caused by chemotherapy in people with low vitamin D levels. About 120 cancer patients will be split into two groups: one gets vitamin D supplements, the other gets standard care. The goal is to see if vitamin D he…

This study tests whether adding balance training, education, and vestibular rehab to the standard Epley maneuver helps people with benign paroxysmal positional vertigo (BPPV) more than the maneuver alone. Forty-six adults with BPPV will receive either the combined approach or sta…

This study tests a spinal cord stimulator (BurstDR) to relieve chronic pain from diabetic neuropathy. Twenty adults with long-lasting leg pain will try a temporary device for one week. If their pain drops by at least half, they can get a permanent implant. The study tracks pain l…

This pilot study aimed to test whether immersive virtual reality (VR) exercises could help people with multiple sclerosis who experience dizziness, balance issues, and fatigue. Participants would have been randomly assigned to either VR-based vestibular rehab or conventional exer…

This study tests a biodegradable tube that is placed over a cut nerve during a leg biopsy. The goal is to see if it is safe and reduces common side effects like pain, redness, and swelling. Twenty adults with various nerve conditions are taking part, and half will get the tube wh…

This study tests whether walking with a robotic exoskeleton three times a week for 12 weeks can reduce nerve pain in people with spinal cord injury. Forty adults with traumatic spinal injury and persistent nerve pain will be randomly assigned to either robotic walking or a relaxa…

This study tests whether a 12-week strength and functional exercise program, done at home via video calls with a physiotherapist, can improve walking, balance, and daily life in people with Hereditary Spastic Paraplegia (HSP). Twenty participants aged 16 to 70 will each experienc…

This study tests whether wearing pressurized gloves and foot caps during chemotherapy can prevent nerve damage (peripheral neuropathy) caused by the drug paclitaxel. It involves 182 people with nasopharyngeal cancer who have not yet started treatment. Each patient serves as their…

This study aims to find the smallest effective dose of the numbing drug ropivacaine for blocking leg nerves during diabetic foot surgery. Researchers will test different concentrations in 45 adults to see which dose works best for 90% of patients. The goal is to provide effective…

This study tests a new drug called LY3848575 for people with long-term nerve pain starting in the feet and moving up the legs. About 558 participants will receive either the drug or a placebo to see if it safely reduces pain. The trial lasts up to 30 weeks and measures pain inten…

This study tests whether giving ketamine during and after surgery can reduce chronic nerve pain in people with traumatic brachial plexus injuries. About 100 adults will receive a ketamine infusion during their reconstructive surgery and for 24 hours afterward. Researchers will me…

This study tests whether a structured exercise program can reduce chemotherapy-induced peripheral neuropathy (CIPN) — nerve damage causing pain, numbness, or tingling. About 133 cancer patients who received certain chemotherapies will either follow an exercise plan or receive sta…

This study aimed to see if a computer-based cognitive training program could improve spatial thinking, balance, and quality of life in people with long-term dizziness from inner ear problems. The training was meant to be used alongside standard physical therapy. However, the stud…

This pilot study tested whether two types of manual therapy (nerve mobilization and neck-based techniques) could help control blood sugar and reduce pain in 75 adults with diabetic peripheral neuropathy. Participants received twice-weekly sessions for 12 weeks, and the study focu…

This study tests a 12-week online program for mothers of infants (12-36 months old) with SMA Type 1. The program aims to teach mothers how to support their baby's development and improve their own knowledge. Thirteen mothers will take part, with 10 receiving the program and 3 rec…

This study is testing whether repeated use of QUTENZA (a capsaicin 8% patch) can reduce pain and the need for painkillers in adults with painful diabetic nerve damage in the feet. About 144 participants will receive up to three patch applications, each 12 weeks apart. Researchers…

This study looks at whether adding a daily 30-minute muscle stimulation session can improve pain control and quality of life for people who already have a spinal cord stimulator (BurstDR) for chronic back pain. Sixty adults with failed back surgery syndrome or persistent spinal p…

This study tests two simple treatments for mild to moderate carpal tunnel syndrome: pain neuroscience education (learning how the brain processes pain) and kinesio taping (applying elastic tape to the wrist). Sixty adults aged 18 to 60 with symptoms for at least 3 months will rec…

This study compares two exercise programs—asynchronous telerehabilitation (remote guidance) and a standard home exercise plan—for people with piriformis syndrome, a condition causing buttock and leg pain. About 46 adults aged 18-45 with confirmed diagnosis will participate. The g…

This study tests whether injecting a 5% dextrose (sugar water) solution around the nerve in the wrist can reduce pain and improve hand function in people with mild to moderate carpal tunnel syndrome. About 96 adults who haven't gotten better with standard care will receive the in…

This study tests a medicated film placed under the tongue to quickly ease severe episodes (autonomic crises) in people with familial dysautonomia. The film contains dexmedetomidine, a drug that calms the body's stress response. Researchers will check if it reduces symptoms like h…

This study tests a self-guided web program called Face-Forward-Web for adults with chronic orofacial pain (lasting over 3 months). The program includes five 30-minute video sessions teaching relaxation, mindfulness, and coping skills. Researchers will enroll up to 30 participants…

This early-stage study tests whether BHV-7000 can reduce chronic pain in people with inherited erythromelalgia, a rare condition causing severe burning pain due to a genetic mutation. Only 5 adults with a specific NaV1.7 mutation will participate, comparing the drug to a placebo …

This study tests whether using a mechanical insufflation device (BiWaze Cough) twice daily for 6 months can slow the decline in cough strength in 20 people with early ALS. Participants will use a facemask or mouthpiece to help inflate their lungs fully before coughing. Researcher…

This study tests whether linaclotide (Linzess) can improve bowel movement frequency in people with type 2 diabetes who have chronic constipation. About 23 participants will receive either the drug or a placebo for two weeks, then switch after a break. The goal is to see if the dr…

This study tests whether using a lower-pressure tourniquet during orthopedic surgeries can reduce pain and the need for opioid painkillers afterward. Standard tourniquets use high pressure, which can cause muscle weakness and pain. The experimental device sets pressure based on t…

This study is tracking over 700 Chinese adults with diabetic peripheral neuropathic pain who are starting mirogabalin. Researchers are monitoring side effects and how well the drug works in everyday medical practice. The goal is to see if mirogabalin is a safe and effective optio…

This study tests whether warm paraffin wax baths or mud therapy (peloidotherapy) can reduce pain and improve hand function in people with mild to moderate carpal tunnel syndrome. Ninety adults will be split into three groups: one gets mud plus home exercises, another gets wax plu…

This study tests whether a drug called istradefylline, combined with short periods of breathing low-oxygen air (acute intermittent hypoxia), can improve breathing in people with ALS. About 40 adults with ALS and healthy volunteers will take part. The goal is to see if this combin…

This study looks at two treatment plans for carpal tunnel syndrome, which causes hand numbness and pain. One plan starts with up to two steroid injections, then offers surgery if needed. The other plan goes straight to surgery. About 258 adults with mild to moderate symptoms will…

This study tests a device called SensoniQ® Treatment Station that uses low frequency soundwaves to prevent or reduce nerve pain caused by chemotherapy in people with gynecologic cancer. The trial includes 80 participants, some starting chemo and others with lasting nerve pain. Re…

This study tests whether low-dose electrical stimulation of the pudendal nerve can improve erectile function in men with mild to moderate erectile dysfunction. Twenty-four men will receive a 20-minute stimulation session, and researchers will measure changes in erection hardness,…

This study compares a 6-month physiotherapy program focused on pelvic floor relaxation to no structured exercise in women with pudendal neuralgia, a chronic pelvic pain condition. Researchers will measure muscle stiffness using ultrasound and track symptoms like pain and sexual d…

This study looks at whether a 6-day course of oral steroids after orthopedic surgery can help reduce pain, nausea, and improve movement. About 500 adults having common surgeries like joint replacement or fracture repair will be randomly assigned to receive either standard care or…

This study tests whether repeated magnetic pulses (rTMS) aimed at the brain's motor area can safely reduce chronic nerve pain that hasn't responded to other treatments. About 32 adults with conditions like post-stroke pain, trigeminal neuralgia, or spinal cord injury will receive…

This study looks at whether massage therapy can reduce nerve problems like pain, numbness, and tingling caused by chemotherapy in people with breast or digestive system cancers. About 90 participants will receive massage sessions to see if it improves symptoms and quality of life…

This study tests a small, implantable device that stimulates nerves to relieve chronic pain after injury or surgery. About 185 adults with peripheral neuralgia will either receive the stimulator plus standard medical care or standard care alone. The goal is to see if the device p…

This study tests an experimental drug called ONO-1110 to see if it can safely reduce pain in people with postherpetic neuralgia, a type of nerve pain that persists after a shingles infection. About 140 Japanese adults with pain lasting at least 3 months will receive either the dr…

This pilot study tests whether using a robotic glove for 20 minutes a day, 5 days a week, can improve quality of life, grip strength, and fine motor control in people with ALS. Five participants will use the glove at home over 8 weeks, with some in-person and telehealth check-ins…

This study tests whether a non-invasive therapy called neurofeedback can help people with chemotherapy-induced nerve pain learn to change their own brain waves to feel less pain. About 80 participants will be assigned to either receive neurofeedback training or a control conditio…

This study tests whether a progressive exercise program can reduce chronic pain in veteran lung cancer survivors aged 60-80. Nine participants will do treadmill walking, resistance bands, and balance exercises. The goal is to see if exercise can be a safe alternative to opioids f…

This study is testing a device called MemorEM, which is a cap that sends low-level electromagnetic waves to the head. The goal is to get feedback from 1,000 patients with neurological diseases like Alzheimer's or Parkinson's, and their caregivers, on how easy the device is to use…

This study tests whether yoga can reduce nerve pain caused by chemotherapy in cancer survivors. About 227 participants will either take yoga classes twice a week for 8 weeks, attend educational sessions, or receive standard care. Researchers will measure pain, balance, fall risk,…

This study tests if acupuncture can reduce nerve pain caused by chemotherapy in cancer survivors. About 255 people who finished chemo at least three months ago will receive either real or sham acupuncture. The goal is to see if this drug-free approach can ease pain and improve da…

This study tests whether a dual-task training program—combining physical exercises like walking with mental tasks like counting—can improve both motor and cognitive function in 16 boys aged 6–12 with Duchenne muscular dystrophy. Participants will be randomly assigned to either st…

This early-phase study tests whether MRI-guided focused ultrasound can safely reduce severe facial pain in people with trigeminal neuralgia that hasn't responded to medication. Ten adults aged 21-75 with chronic pain for over 6 months will receive a precise ultrasound treatment t…

This study is testing the long-term safety and effectiveness of a drug called mexiletine PR for people with myotonic dystrophy types 1 and 2. The drug is taken once daily as a liquid to help reduce muscle stiffness (myotonia). The study includes 176 adults and teens who have alre…

This study tests a daily drug called basimglurant for adults with trigeminal neuralgia, a condition causing severe facial pain often called the 'suicide disease.' The trial includes 166 people who still have pain despite current treatments. After an 8-week run-in on the drug, par…

This study looks at whether a type of electrical current called diadynamic current can reduce pain and improve hand function in people with carpal tunnel syndrome. About 80 adults with mild to moderate carpal tunnel syndrome will receive this therapy along with standard care like…

This study is testing whether a robotic exoskeleton called Atalante can safely help people with ALS maintain their ability to walk. The idea is that the suit provides natural leg movements and feedback to the brain, which may slow down walking decline. About 20 adults with slow-p…

This study compares two types of hands-on therapy—Stecco Fascial Manipulation and Rolfing Structural Integration—for people with piriformis syndrome, a condition that causes buttock and leg pain. Forty-two adults aged 25 to 45 with at least four weeks of pain will receive four we…

This study tests whether a noninvasive electrical stimulation device placed on the skin over the spine can reduce pain and improve quality of life in people with neuropathic (nerve) pain. About 62 adults with chronic low back pain from nerve damage will receive either real or sha…

This small study tests whether eptinezumab, a drug used for migraines, can reduce pain in people with atypical facial pain that hasn't responded to other treatments. Ten participants will receive two intravenous infusions three months apart. The main goal is to see if the drug im…

This study looks at whether a combination of herbal medicine and Chuna manual therapy (a Korean spinal manipulation technique) can safely reduce pain and improve walking ability in people with low back pain or sciatica caused by lumbar spinal stenosis or spondylolisthesis. Resear…

This study looks at adults with chronic pain who receive ketamine infusions at Stanford's Pain Management Center. Researchers want to understand which patients improve and what factors predict a good response. The goal is to better tailor this treatment to individuals.

This study was designed to test a new drug called N0750 against two standard pain medications (Cymbalta and Lyrica) for painful diabetic peripheral neuropathy. It planned to enroll adults with diabetes and nerve pain for at least 3 months. However, the trial was withdrawn before …

This study tests whether pressing specific points on the ear (auricular point acupressure) can reduce pain, numbness, and tingling caused by chemotherapy. About 225 cancer patients will try this non-needle technique. Researchers will also measure changes in nerve function and bod…

This study looks at whether adding dynamic stretching to foam rolling helps people with piriformis syndrome more than foam rolling alone. Forty adults aged 35–50 with buttock pain will do either foam rolling or foam rolling plus dynamic stretches for several weeks. Researchers wi…

This study tests a new, team-based rehabilitation program for 125 working-age adults with persistent physical symptoms and chronic fatigue. The program includes doctors, therapists, and counselors who work together to help patients manage their symptoms and improve daily function…

This study tests whether a new medicine called CagriSema can reduce nerve pain in people with type 2 diabetes. About 142 adults with painful diabetic neuropathy will receive either CagriSema or a placebo for around 10 months. The main goal is to see if the drug lowers weekly pain…

This study compares carbamazepine and oxcarbazepine, two drugs used to treat trigeminal neuralgia, a condition causing severe facial pain. Researchers will enroll 122 adults aged 18-60 who have had the condition for at least two weeks. They will measure pain relief using a standa…

This study tests whether daily exposure to green LED light can reduce chronic pain. 55 adults with conditions like fibromyalgia or nerve pain will use a green or white light device at home for 2 hours each day over 3 months. Researchers will measure changes in pain levels, medica…

This study tests a new way of using spinal cord stimulation to treat long-lasting leg pain caused by diabetic nerve damage. Up to 25 adults with severe pain will first try a temporary device, and if it helps, they can get a permanent implant. The main goal is to see if this appro…

This study tests a precise radiation treatment for trigeminal neuralgia, a condition that causes severe facial pain. Instead of a metal frame screwed into the skull, a special face mask holds the head still while x-rays target the painful nerve. The trial includes 40 adults whose…

This study tests whether neurofeedback training—a non-invasive therapy that uses brain wave monitoring—can help people with chemotherapy-induced peripheral neuropathy (nerve pain, numbness, or tingling) learn to change their own brain activity to feel less pain and improve qualit…

This study tests if a non-invasive treatment called repetitive transcranial magnetic stimulation (rTMS) can reduce nerve pain caused by oxaliplatin chemotherapy in people with stage I-IV cancer. rTMS uses magnetic pulses on the scalp to change brain activity. The trial includes 5…

This study looks at whether a special nerve wrap can help people who need a second surgery for cubital tunnel syndrome, a condition where a nerve in the elbow gets compressed. Twenty adults with ongoing pain or numbness after their first surgery will receive the nerve protector d…

This study tests whether combining two non-invasive therapies—infrared light (MIRE) and electrical nerve stimulation (TENS)—can reduce leg swelling caused by nerve-related blood flow problems. About 1000 adults with lower leg swelling took part. The main goal is to see if the tre…

This study tests two different head-and-body rotation procedures done in a mechanical chair to treat a common inner-ear condition called BPPV, which causes brief but intense spinning sensations. 128 adults with confirmed BPPV will be randomly assigned to either the standard Epley…

This study treated one 58-year-old man with polyneuropathy caused by POEMS syndrome using his own fat-derived stem cells. He received eight infusions over 28 weeks to try to relieve nerve symptoms. The study is no longer available and was not a formal clinical trial.

This study tests whether injecting a patient's own fat (lipografting) into the wrist can improve symptoms of mild to moderate carpal tunnel syndrome better than standard steroid injections. The fat is thought to reduce scarring and help nerves glide more freely. The trial plans t…

This study tests a wearable robotic exoskeleton called EXPLORER in 15 children with movement problems from conditions like cerebral palsy. The goal is to see if it helps them walk better in their everyday environments, such as home and the community. The study focuses on safety, …

This phase II trial tests whether a natural supplement called PEA can relieve nerve pain, numbness, and tingling caused by chemotherapy in cancer patients. About 88 adults who have had nerve symptoms for at least 3 months after chemo will take PEA or a placebo for 8 weeks. The st…

This study tests whether adding sodium bicarbonate (baking soda) to the local anesthetic used during carpal tunnel release surgery reduces the pain of the injection itself. 116 adults with carpal tunnel syndrome will be randomly assigned to receive either buffered or standard ane…

This study compares two common types of stitches used to close the wound after carpal tunnel release surgery: absorbable stitches that dissolve on their own and non-absorbable stitches that need to be removed. About 116 patients will be randomly assigned to one stitch type, and t…

This study tests whether the UpRight Go posture trainer, a wearable device that vibrates when you slouch, can reduce pain, numbness, and weakness in people with cubital tunnel syndrome who haven't had surgery. Researchers at Yale University plan to enroll 40 adults and track symp…

This study tests a non-invasive brain stimulation technique called prolonged continuous theta burst stimulation (pcTBS) to relieve nerve pain caused by chemotherapy in breast cancer patients. Forty women will receive either pcTBS or standard repetitive transcranial magnetic stimu…

This early-stage trial tests the drug dalfampridine (Ampyra), already used for multiple sclerosis, in 35 adults with primary lateral sclerosis or upper motor neuron ALS. The main goal is to see if it safely improves walking speed, measured by a timed 25-foot walk. Researchers wil…

This study planned to test photobiomodulation (a type of light therapy) on nerve pain and sensation in one woman after mastectomy. The treatment used red and near-infrared light on the affected area for six weeks. However, the study was withdrawn before any participants enrolled,…

This study tests a robotic feeding device called Obi for people who cannot use their arms to eat on their own. About 50 participants, including patients, caregivers, and healthcare providers, will use the device at home or in care settings for one week. Afterward, they will fill …

This study tests two types of nerve stretches—active (patient-controlled) and passive (therapist-applied)—to see which better reduces burning pain, improves muscle strength, and increases joint movement in people with diabetic neuropathy. Sixty adults aged 40 to 65 with diabetes …

This study looks at whether people with flat feet (pes planus) are more likely to have piriformis syndrome, a condition that causes pain in the hip and lower back. Researchers will compare foot posture and pain in adults aged 18-65 who have piriformis syndrome with those who do n…

This study looks at two new ways to stage amyotrophic lateral sclerosis (ALS) based on key disease milestones. Researchers will check how well these systems work in 50 patients and see if adding a blood marker called neurofilament can improve tracking of the disease. The goal is …

This study was designed to see if giving a short, mild electrical stimulation to compressed nerves during surgery could speed up recovery of strength and function. It planned to include people with nerve compression in the spine or limbs who were already scheduled for decompressi…

This study is observing 217 people with ALS and related conditions like frontotemporal dementia and hereditary spastic paraplegia. Researchers aim to connect each person's genetic makeup with their symptoms and find biological markers in blood and spinal fluid. No new treatments …

This study looks at people with ALS and similar diseases like frontotemporal dementia and hereditary spastic paraplegia. Researchers want to understand how genes affect the disease and find biological markers (biomarkers) to help develop future treatments. About 708 participants,…

This study enrolls people with ALS and related disorders, as well as healthy volunteers, to track biological markers (biomarkers) in urine, blood, and spinal fluid. The goal is to better understand how these markers change over time, which could help design more effective future …

This 24-month observational study will follow 80 people with Becker muscular dystrophy (BMD) to better understand how the disease progresses. Researchers will measure muscle strength, walking speed, breathing, and heart function using standard tests. The goal is to identify which…

This 24-month observational study follows 100 people aged 12–50 with Limb Girdle Muscular Dystrophy type R1 (LGMD R1). Researchers will test whether a motor function scale called NSAD and muscle fat measurements from MRI can reliably track disease progression. The goal is to vali…

This study tests new radio frequency coils for MRI scanners to see if they can produce better images in children, including those with Duchenne muscular dystrophy. Researchers will check for side effects like heating and discomfort, and measure image quality. The goal is to impro…

This long-term study looks at children and adults with inherited nerve and muscle disorders that start early in life, like muscular dystrophy. Researchers will track symptoms over time and collect genetic samples from affected individuals, their family members, and healthy volunt…

This study planned to see if mindfulness meditation using virtual reality could help people with ALS feel better emotionally and improve their quality of life. It was designed for adults with ALS who speak French. However, the study was withdrawn before enrolling any participants…

This study looks at how people with long-term dizziness (chronic vestibulopathy) walk and keep their balance, comparing them to healthy volunteers. Researchers will measure things like step length, sway, and fear of falling using special equipment and tests. The goal is to better…

This study follows 1000 Chinese patients with myotonic dystrophy type 1 (DM1) to understand how the disease affects multiple body systems and leads to disability. Researchers will collect health data and blood samples every 3 to 6 months for several years. The goal is to identify…

This study follows about 413 adults with multifocal motor neuropathy (MMN) over time to learn how the disease progresses and affects daily life. Researchers will collect information on symptoms, quality of life, and healthcare use during regular doctor visits. No experimental tre…

This study examines how common cardiac autonomic neuropathy (nerve damage affecting the heart) is in children and teens with type 1 diabetes. Researchers will monitor 75 participants using heart tests and check if longer diabetes duration or poor blood sugar control raises the ri…

This study will collect and analyze genetic data from 1000 people with suspected inherited metabolic diseases, including conditions like epilepsy and mitochondrial disorders. Researchers at Karolinska University Hospital aim to improve diagnosis by using advanced genetic testing …

This study looks at whether a tool called the Chelsea Critical Care Physical Assessment tool (CPAx) can accurately track recovery in 120 adults who developed muscle weakness after being on a breathing machine in the ICU. The goal is to find the smallest meaningful improvement tha…

This study looks at whether low blood pressure during major noncardiac surgery harms organs like the heart and kidneys. Researchers are using a non-invasive brain monitor to see if a personalized blood pressure target can be set. 650 patients at cardiovascular risk are being obse…

This study examines whether a low pulse pressure (the difference between top and bottom blood pressure numbers) in the first hours after a cardiac arrest is linked to early death. Researchers will analyze data from 3500 patients who had a cardiac arrest outside the hospital and a…

This study looks at whether diabetes or blood sugar problems can worsen thinking and memory issues in people with myotonic dystrophy type 1. Researchers will use MRI brain scans and thinking tests over 4 years to track changes. The goal is to understand why some patients have mor…

This observational study is looking at a protein called neurofilament light chain (NfL) in the blood of people who carry a gene variant for hereditary ATTR amyloidosis, both those without symptoms and those with nerve damage. Researchers want to see if NfL levels can help detect …

This study tests whether advanced MRI techniques can measure brain changes in people with ALS (Lou Gehrig's disease). Researchers will scan 119 participants three times over 8 months to see if these scans can track the disease and help diagnose it earlier. The goal is to create b…

This study is testing a virtual reality game designed specifically for people with Duchenne muscular dystrophy (DMD). Six participants aged 7 and older will play the game once using a VR headset and hand tracking. Researchers will measure how easy and enjoyable the game is, and w…

This study uses advanced MRI scans and genetic tests to track how neurodegenerative diseases like frontotemporal dementia and ALS spread across the brain's network. Researchers will follow 645 participants, including patients, family members, and healthy controls, for up to 2 yea…

This study looks at whether a simple injection of sugar water (D5W) can reliably bring out a hidden sign of muscle strength in people with drop foot. Researchers will inject the fluid under the skin and check muscle strength before and after. The goal is to see if this test can h…

This study looks at whether anxiety and depression are connected to carpal tunnel syndrome in medical students. Researchers will measure the median nerve in the wrist and check students' anxiety and depression levels using questionnaires. The goal is to see if there is a link bet…

This study checks whether a French version of a scale called PLSFRS works well for people with primary lateral sclerosis (PLS), a rare nerve disease. Researchers will ask 80 patients to fill out the questionnaire during routine check-ups every 3 to 6 months. The goal is to see if…

This study is testing a new way to care for people with rare diseases. It will use advanced genetic testing and a team of specialists to help diagnose patients faster and coordinate their care better. The study involves 136 participants with certain rare diseases and aims to redu…

This study looks at how the COVID-19 pandemic impacted the quality of life of 274 cancer patients who also have neuropathy (nerve pain). All participants had taken part in earlier clinical trials. Researchers want to see if there are differences between those who received a brain…

This study tests whether a specific leg stretching technique, called sciatic nerve mobilization, can change how sensitive the nerves in your lower leg are to pressure pain. 34 healthy university students will either receive the stretch or no treatment. Researchers will measure pa…

This study will measure nerve signals in 50 adults aged 45 to 76 with diabetic peripheral neuropathy. Researchers will compare these signals with blood sugar levels, diabetes duration, and neuropathy symptom scores. The goal is to find better ways to detect nerve damage early.

This study tests whether giving patients with hand problems like carpal tunnel syndrome or trigger finger a decision aid—a booklet with clear information about their condition and treatment options—helps them feel more satisfied and less conflicted about their choices. About 126 …

This study is checking whether a questionnaire called the MAP-Hand scale is a valid and reliable way to measure hand activity in people with carpal tunnel syndrome. Researchers will enroll 180 adults with mild or moderate carpal tunnel syndrome and compare their scale results to …

This study looks at how the nervous system becomes overly sensitive in people with different types of chronic pain. Researchers will assess 185 adults using questionnaires, thinking tests, and laser brain-wave measurements at the start and after one year. The goal is to better un…

This study tests a lifestyle program for African American men who have finished prostate cancer treatment and their spouses or partners. The goal is to see if the program can improve quality of life, diet, physical activity, and partner support. Only 6 participants were enrolled,…

This study involves 324 adults with facioscapulohumeral muscular dystrophy (FSHD) across 14 international sites. Researchers are testing two new measurement tools—a functional test called FSHD-COM and a muscle-impedance device—to better track disease progression. The goal is to i…

This study was designed to observe brain wave (EEG) changes in adults with chronic neuropathic pain during ketamine treatment. Researchers hoped to find patterns that could predict which patients benefit most. However, the study was withdrawn before enrolling any participants, so…

This study measures how often people with myotonic dystrophy type 1 fall and what factors affect their balance. Researchers will also assess fear of falling and leg muscle strength. The goal is to better understand fall risk in this condition.

This study looks at how combining mirror therapy, electrical stimulation, and motor imagery changes brain activity in people with recent forearm nerve injuries and in healthy adults. Researchers want to understand if these therapies can help the brain adapt better after injury. T…

This study looks at people with early ALS who still have some unaffected body areas. Researchers want to find the very first signs of the disease spreading. By observing 100 participants over time, they hope to learn how ALS starts and progresses, which could help with earlier di…

This study aims to find early biological markers that can predict muscle weakness in children who need a breathing machine in the intensive care unit. About 500 children will be observed to see who develops this condition. The goal is to create a warning system so doctors can int…

This study looks at how much energy critically ill patients on ventilators use during routine passive physiotherapy. By measuring oxygen consumption with a special device in the breathing tube, researchers hope to understand the physical demands of these exercises. The goal is to…

This study is developing an early warning model to predict ICU-acquired weakness in children who need mechanical ventilation. Researchers will track 1,500 children in the PICU, looking for biomarkers and other signs that could forecast muscle weakness. The goal is to move from di…

This study follows 30 infants with congenital heart disease who need heart surgery. Researchers will use blood tests, EEG brain wave monitoring, and MRI scans to look for signs of brain injury. The goal is to find better ways to detect brain damage early and identify which childr…

This study looks back at the medical records of about 4 million people who had physical or occupational therapy for muscle and joint problems. Researchers want to see if different ways of giving therapy lead to different results. No new treatments are tested—the goal is to learn …

This study aimed to see if using stitches that dissolve (absorbable) or stitches that need removal (non-absorbable) leads to better scar appearance and patient satisfaction after carpal tunnel or trigger finger surgery. It was planned for adults having these common hand surgeries…

This study looks at how the muscles in the arms and hands change in people with Duchenne muscular dystrophy (DMD). Researchers will measure muscle tone, stiffness, and elasticity using a device called MyotonPRO. They will also test hand function and muscle strength. The goal is t…

This study looks at people with trigeminal neuralgia, a condition that causes severe facial pain. Researchers want to see if adding virtual reality (VR) training before Gamma Knife surgery can improve pain relief. They will also use artificial intelligence (AI) and brain scans to…

This study looks at how thinking skills (called executive functions) relate to everyday activities like self-care and play in children with Duchenne muscular dystrophy (DMD). Researchers will compare 38 children with DMD to healthy children using surveys and functional tests. The…

This study looks at whether common balance tests can tell doctors why a person is having trouble with balance. Researchers will test 330 people, including healthy volunteers and patients with stroke, vision problems, inner ear issues, spine problems, or spinal cord damage. The go…

This study follows about 400 people with Duchenne muscular dystrophy who have already received the gene therapy SRP-9001 in an earlier study. The goal is to monitor their safety and muscle function over time. No new treatment is given in this study.

This study is tracking 2000 adults with carpal tunnel syndrome who are having surgery to release the nerve. The surgery uses a special device called UltraGuideCTR, which is guided by ultrasound. Researchers are collecting real-world data on symptoms and hand function for up to 24…

This study looks at how common nerve problems are in people who feel eye discomfort. Researchers will test 1,000 participants to see if their pain is linked to nerve damage rather than just dry eye. The goal is to better understand these conditions and improve diagnosis.

This study follows 46 people with gyrate atrophy, a rare genetic condition that causes vision loss, over 4 years. Researchers measure ornithine levels in the blood and track changes in the retina using eye scans and photos. The goal is to learn how the disease progresses under st…

This study tests a new ultrasound technique called shear wave elastography to measure stiffness in the wrist nerves and tissues of people with carpal tunnel syndrome. Researchers will compare these measurements with standard tests before and after treatment (surgery or steroid in…

This study follows 25 people with limb-girdle muscular dystrophy type 2A (LGMD2A), a rare genetic disease that causes progressive muscle weakness. Researchers will measure how muscle strength changes over time and how it affects quality of life. The goal is to better understand t…

This study collects blood or skin samples from 100 adults with inherited heart rhythm disorders (like Long QT Syndrome or Brugada Syndrome) and healthy volunteers. Researchers will turn these samples into stem cells and then into heart cells to study how these diseases work and t…

This study follows 200 African American prostate cancer survivors and their partners to understand how daily stress, social support, and lifestyle choices like physical activity and diet affect each other. Participants wear an activity tracker and answer surveys on their smartpho…

This study looks at how well a new test called quantitative sensory testing (QST) can measure nerve function in the eye. Researchers will use heat, cold, and vibration to see how people with dry eye disease or corneal nerve damage respond. The goal is to better understand eye pai…

This study aims to discover new genetic mutations that cause mitochondrial disorders by analyzing tissue samples from up to 6,900 participants. It includes people with suspected or known mitochondrial diseases, such as MELAS or Leigh's Disease, who lack a genetic diagnosis. The r…

This study follows 20 adults with hereditary transthyretin amyloidosis (ATTRv) who have both nerve and heart involvement. They are treated with patisiran or vutrisiran for two years. Researchers use MRI scans and questionnaires to see how the drugs affect heart structure and func…

This study aims to help doctors diagnose neuromuscular diseases faster and better understand how these conditions progress. Researchers will collect blood, tissue, and imaging data from 500 adults with neuromuscular disease. The goal is to find genetic markers and other clues tha…

This study is collecting MRI scans from 1000 people with neuromuscular diseases to see if muscle imaging can help doctors diagnose these conditions more accurately and without invasive biopsies. The goal is to create a large database of MRI patterns linked to specific genetic cau…

This study aimed to find out how many people with trigeminal neuralgia (a severe facial nerve pain condition) also have temporomandibular disorder (TMD), which causes jaw pain. Researchers planned to follow patients from three clinics and test a questionnaire to better diagnose b…

This study looks at whether a new care pathway can shorten wait times for people with carpal tunnel syndrome. Researchers will compare wait times before and after the pathway is put in place. About 200 adults will take part and fill out a short satisfaction survey.

This study follows 1000 people with upper motor neuron disease who are about to receive the Cionic Neural Sleeve. The sleeve uses electrical stimulation to help muscles contract at the right time during movement. Researchers will track changes in health-related quality of life us…

This study is working to create better tools for measuring muscle function in people with facioscapulohumeral muscular dystrophy (FSHD). Researchers will follow 100 adults with FSHD for up to 24 months, testing new ways to track disease progression. The goal is to make future cli…

This study is testing a new, comfortable headset that reads brain signals to help people with ALS type on a virtual keyboard. The goal is to improve the device so it can be used in daily life. Five people with ALS will try the headset, and researchers will measure how easy it is …

This study was designed to track heart muscle changes in people with Duchenne muscular dystrophy using two cardiac MRIs taken two years apart, along with blood tests for heart failure markers. It planned to enroll participants aged 6 and older with a confirmed genetic diagnosis. …

This study looks at 150 people with knee osteoarthritis to see how often night pain and nerve pain (neuropathic pain) occur together. Researchers will use questionnaires and physical exams to measure pain and sleep quality. The goal is to better understand these pain types and th…

This study follows 205 people with four types of limb-girdle muscular dystrophy (LGMD) to understand how the disease changes over time. Participants will have their muscle strength, movement, and breathing tested regularly for up to 5 years. No treatment is given; the goal is to …

This study was designed to track the natural course of gamma-sarcoglycanopathy (LGMDR5), a rare muscle-weakening disease, over two years. Researchers planned to measure changes in muscle strength, walking ability, and daily function in patients aged 6 to 35. The goal was to bette…

This study aims to find better ways to measure disease progression in people with advanced facioscapulohumeral muscular dystrophy (FSHD) who use wheelchairs. Researchers will test various muscle strength and function tests over two years in 30 adults. The goal is to develop relia…

This study looks at whether a power wheelchair that can lift a child from sitting to standing is practical for kids with conditions like cerebral palsy, spinal cord injuries, or genetic diseases. Only 4 children aged 5-17 are taking part. The goal is to see if the chair helps the…

This study follows 35 infants with spinal muscular atrophy (SMA) who are receiving gene therapy or other advanced treatments. Researchers use small wearable motion sensors to measure how well the babies move their arms and legs over two years. The goal is to create a more precise…

This study looks at 80 people with ALS to see if electrical tests (EMG) and ultrasound scans of breathing muscles can predict when they will need breathing support. The goal is to start noninvasive ventilation at the right time. Researchers will also check for links between muscl…

This observational study will follow 300 people with Duchenne muscular dystrophy who are already taking exon-skipping therapies (eteplirsen, golodirsen, or casimersen) as part of their routine care. Researchers will collect data on movement, lung function, and heart function over…

This observational study will enroll 40 adults with facioscapulohumeral muscular dystrophy (FSHD) to understand how muscle degeneration affects movement. Researchers will use MRI to assess muscle health and 3D motion capture to analyze walking and posture. The goal is to create a…

This study is a registry that collects information on up to 600 children in China with spinal muscular atrophy (SMA). It aims to describe how the disease progresses and how treatments are used in everyday medical practice. The study does not test a new drug but gathers data to be…