Familial amyotrophic lateral sclerosis

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 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 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 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 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 study is testing a smartphone app that records and analyzes speech to help diagnose and monitor neurodegenerative conditions like dementia, Parkinson's disease, multiple sclerosis, and motor neuron disease. Researchers will compare speech patterns from 150 people with these …

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 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 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 aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

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

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

This study aims to 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 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 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…