Anterior horn disorder

Researchers are testing whether a new oral polio vaccine called nOPV2, given at birth, helps infants block type 2 poliovirus in their intestines. The trial enrolls 740 healthy newborns who have received standard injected polio vaccines. Scientists will check stool samples for sig…

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

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

This phase 4 study compares a single dose of an inactivated polio vaccine (sIPV) in people aged 7 to 50 years with the standard three-dose series given to infants. Researchers want to see if one shot can produce a similar immune response. The trial is recruiting 180 healthy parti…

This study tests a new oral polio vaccine (nOPV1) in over 2,200 healthy young children, infants, and newborns in Bangladesh. Researchers will compare it to the current Sabin vaccine to see if it is safe and triggers a strong immune response. The goal is to find a more stable vacc…

This study tests whether giving two new oral polio vaccines (types 1 and 2) together is safe and works as well as giving them separately in healthy 16-week-old infants. About 675 babies in Panama will receive either the combo or single vaccines. The goal is to see if the combo ap…

This study will follow 36 people with spinal muscular atrophy (SMA) who receive a gene therapy called onasemnogene abeparvovec (ITVISMA) injected into the spinal fluid. The goal is to see how well it works and how safe it is in everyday medical practice. Participants must be at l…

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

This study tests an experimental drug called NB-4746 in 80 people with ALS. The goal is to see if it is safe, how it moves through the body, and whether it can reduce a marker of nerve damage (NfL) in the blood. Participants are randomly assigned to receive either NB-4746 or a pl…

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

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

This study tests a drug called apitegromab in children under 2 years old who have spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The drug works by blocking a protein that limits muscle growth. Researchers want to see if adding apitegromab to stand…

This study tests an experimental drug called ALN-SOD in people with ALS caused by a mutation in the SOD1 gene. The drug is given via spinal injection to target the root cause of the disease. The main goal is to check safety, but researchers will also measure effects on biomarkers…

This early study tests a device called MyoRegulator® that uses mild electrical currents to calm overactive motor neurons in people with ALS. The goal is to see if it is safe and feasible, and whether it might slow disease progression. Fifteen participants will receive the treatme…

This early-stage study tests a new drug called VTx-002 in 12 people with ALS. The main goal is to check if the drug is safe and tolerable when given as a single injection into the fluid around the brain and spinal cord. Researchers will also look for signs that the drug might slo…

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

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

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

This study looks at how the body handles the drug nusinersen when given through a new implantable device called ThecaFlex DRx™, compared to the usual method of a lumbar puncture (spinal tap). About 58 people with spinal muscular atrophy (SMA) who are already in another study (PIE…

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

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

This 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 is testing a single injection of a gene therapy called INS1202, given into the spinal fluid, for people with ALS (Lou Gehrig's disease). The study includes 23 adults, some with a specific genetic mutation (SOD1) and some without known genetic causes. The ma…

This early-phase trial tests a gene therapy called SKG0201 in 11 infants with spinal muscular atrophy (SMA) type I, a severe muscle-weakening disease. The therapy uses a harmless virus to deliver a working copy of the missing SMN1 gene. The study aims to see if it is safe and can…

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

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

This trial tests a new implantable device called ThecaFlex DRx in 90 people with spinal muscular atrophy (SMA) who cannot receive standard spinal injections due to spine problems or other risks. The device is placed under the skin and delivers the medication nusinersen directly i…

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

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

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

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

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

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

This study tests whether a device called the LIC Trainer, used twice daily at home, can slow the decline in lung function and delay the need for a breathing machine in people with ALS. Fifteen adults with early-stage ALS will use the device and be monitored every three months. Re…

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

This phase 2 trial is testing whether an inhaled drug called cromolyn (PHENOGENE-1A) can help people with mild-to-moderate ALS when added to standard care. About 105 participants will receive either a low dose, high dose, or placebo for 24 weeks. Researchers will measure changes …

This expanded access program offers the drug tazbentetol to adults with ALS who cannot join other clinical studies. Participants take one 300 mg pill by mouth each day. The program aims to check the drug's safety and whether it helps control the disease.

This pilot trial tests whether transplanting gut bacteria from healthy donors (MTP-101C) is safe and can help slow ALS. Twenty people with fast-progressing ALS will receive the treatment after a bowel cleanse. Researchers will track changes in muscle function, quality of life, an…

This study tests a single dose of GB221, a gene therapy that delivers a working SMN1 gene, in infants aged 2 weeks to under 12 months with spinal muscular atrophy (SMA) Type 1. The goal is to see if it is safe and helps improve motor function. The trial includes both symptomatic …

This study tests whether starting non-invasive ventilation (a breathing machine) earlier than usual helps people with ALS. Researchers will use a prediction tool to find those at high risk of breathing problems within 6 months. 48 participants will be randomly assigned to early N…

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

This phase 2 trial tests whether tofersen, a drug already approved for a specific type of ALS, can help people with ALS who do not have the SOD1 gene mutation. Thirty adults will receive the drug via spinal injections over 24 weeks. Researchers will measure changes in nerve damag…

This early-stage trial tests a brain-computer interface called BrainGate for people with paralysis from conditions like ALS or spinal cord injury. A small sensor is placed in the brain to interpret movement-related signals, allowing users to control a tablet computer just by thin…

This study tests an experimental drug, dazucorilant, in 279 adults with ALS (Lou Gehrig's disease). The goal is to see if the drug can slow the loss of muscle function and improve quality of life over 24 weeks. Participants will receive either the drug or a placebo, and researche…

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

This study tests the safety and feasibility of the BrainGate2 system, a brain-computer interface. Tiny sensors are placed in the brain area that controls movement, allowing people with tetraplegia (paralysis of all four limbs) to control a computer cursor or other assistive devic…

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

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

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

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

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

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

This early-stage trial tests a new medicine, AMX0114, in 48 adults with ALS. The drug aims to reduce a protein called calpain-2, which may damage nerves. The main goal is to check safety and how the body handles the drug, not yet to prove it works.

This early study tests a brain-computer implant that reads brain signals to help people with severe paralysis (from ALS, stroke, or spinal cord injury) communicate. The device aims to decode imagined speech and allow control of computers. Only 2 participants will be enrolled to c…

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

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

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

This early-stage study tests a new drug called LY4256984 in 32 people with sporadic ALS. The drug is injected into the spine to see if it is safe and how the body handles it. The main goal is to check for side effects, not yet to prove it works.

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

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

This early study tests the safety and function of a brain-computer interface (BCI) implanted by a robot in people with severe paralysis. The device aims to let users control external devices like computers or phones using their thoughts. The trial enrolls 15 adults with quadriple…

This study is testing a gene therapy called vesemnogene lantuparvovec for spinal muscular atrophy (SMA) in low- and middle-income countries. Researchers will track safety and whether children reach motor milestones like sitting or walking. The trial involves 15 participants and a…

This early-stage trial tests whether injecting lab-grown nerve cells (made from stem cells) into the spinal cord is safe for people with ALS, a fatal disease that destroys movement control. Twelve participants will receive the cells either once or four times over six months. The …

Neuralink is testing a brain-computer interface called the N1 Implant, placed by a robot, to help people with severe paralysis communicate. The study will enroll 6 adults with conditions like ALS or spinal cord injury who cannot speak or use their hands well. The main goal is to …

This study tests a brain implant that records brain signals to help people with locked-in syndrome control a computer. Two participants will have 128 electrodes placed on their brain surface. Researchers will train them to use the system at home for one year, aiming for accurate …

This study tests a new approach for people with motor neuron disease (ALS) that combines personalized brain stimulation with mixed reality exercise games. The goal is to slow disease progression and improve quality of life. One hundred adults with mild to moderate ALS will be ran…

This early-stage trial is testing whether a special type of immune cell, called regulatory T cells (Tregs), is safe for people with ALS. The cells are taken from each patient's own blood, multiplied in a lab, and then injected into the spinal fluid. The study will enroll 12 adult…

This study tests whether acetyl-L-carnitine (ALCAR), a natural supplement, can slow the loss of self-sufficiency in people with ALS. About 246 adults with early-stage ALS will receive either a low or high dose of ALCAR or a placebo for 48 weeks, alongside standard care. Researche…

This Phase 3 trial is testing whether adding masitinib to the standard ALS drug riluzole can slow the disease better than riluzole alone. About 495 people with ALS will take either masitinib or a placebo, plus riluzole, and researchers will track changes in muscle function, breat…

This phase 2 trial tests whether darifenacin, a drug currently used for overactive bladder, can help people with ALS. The study will enroll 30 adults aged 18-85 with confirmed ALS. Participants will take either darifenacin or a placebo daily for 24 weeks. The goal is to see if th…

Researchers at Johns Hopkins University are studying whether neuromuscular ultrasound and MRI can serve as reliable markers of swallowing muscle problems in people with inclusion body myositis (IBM). The study compares imaging and clinical findings in 30 participants, including p…

Motor neuron disease (MND), including ALS, is hard to diagnose and often takes years to confirm. This pilot study uses a powerful 7 tesla MRI scanner to look for detailed brain features, called imaging biomarkers, that are specific to MND and its subtypes. Researchers will scan 3…

This early-phase trial is testing whether an experimental imaging agent called [18F]ACI-19626 can safely and reliably detect abnormal clumps of a protein called TDP-43 in the brain using PET scans. TDP-43 buildup is linked to certain forms of dementia, such as frontotemporal deme…

This study is working on a new blood test for pregnant women that can check for serious genetic conditions like cystic fibrosis, spinal muscular atrophy, and sickle cell disease. Researchers will collect blood samples from 4,000 pregnant women who are at higher risk of passing on…

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

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

This study is testing whether a quick, painless measurement of muscle activity using surface electrodes can help doctors diagnose ALS earlier and more accurately. Researchers will compare this test to current methods in 650 people with muscle symptoms. They will also track how th…

This study tests whether measuring a specific receptor (P2X4) on blood cells can help diagnose amyotrophic lateral sclerosis (ALS), a severe nerve disease. Researchers will compare P2X4 levels in 50 people — some with ALS and some healthy volunteers. If the test works, it could l…

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

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

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

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

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

This study tests if ranolazine, a heart medication, can safely reduce muscle cramps and improve quality of life in people with ALS. About 72 adults with ALS who have frequent cramps will receive either the drug or a placebo for 28 weeks. The main goals are to check safety and see…

This study tests whether a single 30-minute session of transcutaneous electrical nerve stimulation (TENS) on the face can reduce jaw and facial pain, improve jaw movement, and make chewing easier for people with ALS or PLS. Ten participants will receive the treatment during one c…

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

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

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

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

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

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

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

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

This study looks at whether adding a specific pressure setting (PEP) to a standard cough-assist machine can help people with ALS cough more effectively. ALS weakens the muscles needed to cough, raising the risk of lung infections. The trial will compare cough strength with and wi…

This study tests whether a personalized physical therapy program, designed based on each patient's specific muscle strengths and weaknesses using a robotic leg press, can help people with ALS maintain physical function better than standard physical therapy. Thirty-eight participa…

This study tests a new home-based program that uses a special treadmill and virtual reality to help children with conditions like cerebral palsy, muscle diseases, or obesity improve their walking. About 30 children aged 6 to 17 will try the program at home for at least 3 sessions…

This study tests two types of breathing exercises together in people with ALS. The goal is to see if these exercises can improve cough strength and breathing, and reduce shortness of breath. Up to 34 people with ALS will participate, and the study also looks at how the exercises …

This study tests whether a video-based home exercise program can improve trunk and limb control and daily activities in 20 people with ALS. Participants will follow the program 3 days a week for 8 weeks. The goal is to see if this approach can ease symptoms and improve quality of…

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

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

This study creates a registry of pregnancies where the fetus received a genetic diagnosis of spinal muscular atrophy (SMA) and was treated with a disease-modifying therapy while in the womb, such as risdiplam. Researchers will collect information from medical records and question…

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

This study follows 600 people with neurodegenerative diseases such as Parkinson's, Alzheimer's, and related conditions over 1 to 10 years. Researchers collect data from routine clinical exams, brain imaging, movement analysis, and blood tests. They use statistical tools and artif…

Researchers at the University of Pennsylvania are building a large repository of blood, DNA, and family history data from people with age-related brain diseases like Alzheimer's, Parkinson's, and ALS, as well as their family members. The goal is to support studies into the geneti…

Researchers at Mayo Clinic are collecting blood samples and clinical information from people with ALS, their family members, and healthy controls. The goal is to create a large repository of genetic material that scientists can use to study how genes contribute to ALS. This study…

This study is gathering medical information, blood samples, and speech recordings from 2,000 people with ALS and healthy volunteers over two years. The goal is to create a shared resource that scientists can use to better understand ALS and develop new treatments. Participants ca…

This study follows 600 adults who are genetically at risk for ALS due to family history. Over three years, researchers collect blood samples, speech recordings, and health data to track early signs of the disease. The goal is to build a rich database that scientists can use to be…

This trial tests a brain-computer interface called CortiCom, which is surgically placed on the brain's surface to record signals related to speech and arm movement. The goal is to see if these signals can be decoded to control software and devices, potentially helping people with…

This study is building a registry of people with spinal muscular atrophy (SMA) in the UK and Ireland. Participants complete questionnaires about their symptoms, motor function, medications, and family history. The goal is to gather detailed information to better understand the di…

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

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

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

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

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

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

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

This study watches how spinal muscular atrophy (SMA) drugs change muscles and nerves in 24 children aged 5 to 20. Using MRI scans, strength tests, and blood samples, researchers track changes over a year. The goal is not to test a new treatment but to better understand how existi…

This study collects tissues and MRI scans from 40 adults with ALS to find markers of disease progression. Researchers will compare imaging and genetic data from spinal cord and brain areas. The goal is to better understand ALS and guide future research.

This study aims to find early markers for ALS by analyzing blood, urine, spinal fluid, tears, and smell tests in 60 people. It includes those with ALS gene mutations but no symptoms, early ALS patients, and healthy controls. The goal is to enable earlier diagnosis and better trea…

This study is for people diagnosed with ALS, MND, or PLS. Participants fill out an online survey once a month about their physical function, diet, supplements, medications, and mental outlook. The goal is to see which therapies and behaviors are linked to better outcomes. No new …

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

This study gathers information from the medical records of 1,200 people with ALS and similar conditions. Researchers will use this data to better understand the disease and improve future studies. No experimental treatments are given—the goal is simply to learn from real-world pa…

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

This early-stage study tests a brain implant called BrainGate in just 3 people with severe paralysis or speech loss from conditions like ALS or spinal cord injury. The goal is to see if the device is safe and can let users control a computer cursor or other tools by thought alone…

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

This study is building a large database of health information from 5,000 people with ALS and other motor neuron diseases. Participants share details like symptoms, test results, and disease progression during regular clinic visits. The de-identified data is then shared with resea…

This study is testing whether a smartphone app can accurately measure how people walk, without needing special markers or equipment. Researchers will compare the app's measurements to a standard motion-capture system in 30 volunteers—some with neuromuscular diseases like SMA or m…

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

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

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

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

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

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

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

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

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

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

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

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

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

This study looks at the long-term safety of the drug tofersen (Qalsody®) in people with a rare, inherited form of ALS called SOD1-ALS. Researchers will collect health information from about 125 participants in Europe and the US over at least 7 years. The main goal is to see what …

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

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

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

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

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

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

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

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

This study gathers health information from pregnant women with spinal muscular atrophy (SMA) who have taken the drug Spinraza (nusinersen), and from their babies. Researchers want to learn about pregnancy outcomes, such as miscarriage, birth defects, and infant development. The s…

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

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

This early-stage study tests a new drug called amisodin in 48 healthy adults to see if it is safe and how the body processes it. The study is a first step before testing the drug in people with ALS. Participants receive either the drug or a placebo, and researchers monitor for si…

This study at Penn State University will have 1,300 listeners judge recordings of speech from people with ALS and healthy speakers. The goal is to see how different conversation tasks affect how clearly someone with ALS is understood. No treatment is involved; this is purely obse…

This study uses non-invasive eye scans (OCT and OCTA) to look for changes in the retina that might be linked to brain diseases like Alzheimer's, Parkinson's, and multiple sclerosis. Researchers aim to find early markers that could help diagnose or track these conditions. The stud…

This study uses surveys to learn about the care, needs, and quality of life of people with ALS who have the C9orf72 mutation, as well as their caregivers. Researchers want to find out what support is missing and how to improve daily life for both groups. About 208 participants wi…

This study tests a new PET scan tracer to measure levels of a protein called Hsp90 in the brain. Hsp90 helps proteins fold correctly, and problems with this process are linked to Alzheimer's, Parkinson's, and ALS. Researchers will scan 48 healthy volunteers and patients to see if…

This study follows 60 children with spinal muscular atrophy (SMA) who are already receiving SMN-restoring therapies like nusinersen or gene therapy. Over two years, researchers will track their muscle function, fatigue, breathing, and thinking skills to describe new disease patte…

This study collects information and samples from 1,000 people with or at risk for brain diseases like Alzheimer's, ALS, and frontotemporal degeneration. Researchers will track changes in thinking, language, and brain scans over time. The goal is to build a resource for future stu…

This study aims to find better ways to measure fatigue and walking problems in people with neuromuscular diseases like muscular dystrophy and spinal muscular atrophy. Researchers will use a wearable sensor to track physical activity for one week in daily life and during a walking…

This study collects blood and optional spinal fluid samples from 350 people with Parkinson's, ALS, glioblastoma, certain cancers, or rheumatoid arthritis. The goal is to find new genetic markers that could lead to better diagnostic tests for autoimmune encephalitis and paraneopla…

This study is a registry that collects health information from people in Switzerland who have neuromuscular disorders like SMA, DMD, BMD, and others. It aims to track symptoms, treatments, and outcomes over time to help researchers and doctors improve care. No new treatments are …

This study looks at the experiences of 200 adults with spinal muscular atrophy (SMA) to understand how they were diagnosed and treated. Researchers want to find out why some patients are not receiving approved therapies, such as financial or access barriers. The goal is to improv…

This study tests a new way to read DNA that may find hidden carriers of spinal muscular atrophy (SMA). Some people carry two copies of the SMN1 gene side by side, making them carriers that standard tests miss. Researchers will take blood samples from 27 adults and use long-read D…

This study aims to find biological markers in the blood that can help measure whether a potential ALS therapy is working. Researchers will collect tiny particles shed by brain cells from 30 ALS patients and 30 healthy volunteers. By comparing these particles, they hope to identif…

This study enrolls family members of people with inherited ALS who do not yet have symptoms. Participants receive genetic counseling and testing, and are followed yearly to track when and how symptoms might appear. The goal is to gather information that could one day lead to trea…

This study follows 140 adults with ALS for up to 3 years, collecting spinal fluid and blood samples along with clinical and cognitive tests. The goal is to understand how certain biological markers relate to different forms of the disease and its progression. This is an observati…

This study collects blood and spinal fluid samples from 100 people with ALS or similar neurodegenerative disorders. The samples are stored for future research to find biomarkers that could help understand or diagnose these diseases. Participants include patients and their spouses…

This study uses special wearable devices to monitor how babies and children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy (DMD) move at home. About 100 children will wear a sensor-filled jumpsuit or an ankle monitor to track their motor skills over up to 30 mo…

This study is collecting nerve samples and biofluids from 400 people with ALS or peripheral neuropathies to find biological markers for early diagnosis and disease tracking. By analyzing individual cells, researchers hope to identify molecular changes that happen before symptoms …

This study is for people with ALS, a disease that weakens muscles over time. Researchers want to see if a small wearable sensor called Actimyo can measure walking and arm movements during daily life. About 20 participants will wear the sensor for one month after each clinic visit…

This study uses powerful MRI scans to find biomarkers—measurable signs of disease—for amyotrophic lateral sclerosis (ALS) and primary lateral sclerosis (PLS). Researchers will compare brain images from patients and healthy volunteers to better understand these diseases and potent…

This study tests a brain implant that reads signals from the brain to help people who have lost the ability to speak fluently communicate through a computer just by trying to talk. It involves up to 2 participants with conditions like spinal cord injury or ALS. The main goal is t…

This study aims to learn how the binding of RNA with DNA (called R-loops) is linked to amyotrophic lateral sclerosis type 4 (ALS4) and other inherited neurological disorders. Researchers will observe up to 330 people aged 5 and older, including those with ALS4, related conditions…

This study aims to find new biological markers for neurodegenerative diseases like Alzheimer's, Parkinson's, and ALS. Researchers will analyze genetic, protein, and metabolic data from 1,200 patients to improve diagnosis, predict disease course, and monitor treatment response. Th…

This study looks at how spinocerebellar ataxia (SCA) affects the eyes and vision. Researchers will examine 60 adults with SCA types 1, 2, 3, or 27B, including those with early symptoms and those who are not yet symptomatic. The goal is to measure vision changes and eye damage usi…

This study aims to improve how doctors assess movement in infants with spinal muscular atrophy (SMA), a severe muscle-weakening disease. Researchers will use sensors to measure babies' movements in a special bathtub, where water helps reduce gravity's effects. The goal is to crea…

This study is creating a large collection of blood, spinal fluid, and urine samples from 800 ALS patients and 200 healthy volunteers. Researchers will also track clinical changes and at-home measures over time. The goal is to provide a valuable resource for scientists worldwide t…

This observational study will follow 1,000 people with spinal muscular atrophy (SMA) in Spain over many years. Researchers will collect information on motor skills, breathing support, and feeding needs during routine clinic visits. The goal is to standardize how SMA data is gathe…

This study uses a special PET scan to measure tau protein buildup in the brains of people with frontotemporal lobar degeneration (FTLD), atypical Alzheimer's disease, and healthy volunteers. Researchers want to see if this scan can help tell these conditions apart. About 72 adult…

This study uses powerful MRI scanners to look at the brains and spinal cords of 700 people, including healthy volunteers and those with conditions like multiple sclerosis, Alzheimer's, and Parkinson's. The goal is to understand why some brains stay healthy while others decline. R…

This study follows 3,000 childhood cancer survivors in Switzerland to learn about long-term health problems caused by cancer treatments. Participants undergo physical exams, lab tests, and questionnaires during regular follow-up visits. The goal is to better understand and manage…

This study is creating a national registry of 100 people with ALS in Thailand. Researchers will collect data on symptoms, genetics, and survival to better understand the disease. The goal is to build a foundation for future research and improve care for ALS patients.