SPINAL MUSCULAR ATROPHY

This study follows about 400 adults and children with spinal muscular atrophy (SMA) who are taking the medication risdiplam (Evrysdi). Researchers will monitor participants for about 2 years to see how safe and effective the drug is over the long term. The goal is to better under…

This study looks at whether a higher dose of nusinersen can improve arm and hand function in teenagers and adults with spinal muscular atrophy (SMA) who cannot walk and have already taken another SMA drug called risdiplam. Participants receive two high loading doses, then regular…

This study follows 20 adults with spinal muscular atrophy (SMA) type II who are already taking SPINRAZA, a drug given via spinal injection. Researchers will measure changes in motor function, like sitting, standing, and arm movement, over 27 months. The goal is to see if the drug…

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

This study tests an experimental medicine called NMD670 in 54 adults with type 3 spinal muscular atrophy, a condition that causes muscle weakness. Participants must be able to walk at least 50 meters without help. The goal is to see if the drug improves walking distance and muscl…

This study looks at whether a 12-week online program can help mothers of toddlers (12-36 months old) with spinal muscular atrophy (SMA) type 1 learn more about child development and improve their child's skills. The program includes weekly 90-minute sessions. Researchers will mea…

This study aims to create a simple test to determine the risk of falling for people with neuromuscular disorders like muscular dystrophy, ALS, and myasthenia gravis. Researchers will evaluate how well a series of physical tasks (like standing up, walking, and balancing) can predi…

This study looks at whether a powered wheelchair that lets children stand up can help them take part more in everyday activities. It includes children aged 5-17 with conditions like cerebral palsy or spinal cord injuries who cannot walk. The goal is to see if this approach is pra…

This study aims to better measure how well babies with spinal muscular atrophy (SMA) can move after receiving new treatments like gene therapy. Researchers will use small motion sensors on the babies' arms and legs to track changes in movement over time. The goal is to develop a …