SPINAL MUSCULAR ATROPHY (SMA)

This study is testing a gene therapy called vesemnogene lantuparvovec for people with spinal muscular atrophy (SMA) in low- and middle-income countries. The goal is to see how well it works and how safe it is in everyday medical practice. About 15 participants will be followed fo…

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

This study tests a new medicine, ARGX-119, in children aged 5 to 17 with spinal muscular atrophy (SMA). The goal is to find the right dose and check if it is safe and helps improve movement. All children will continue their usual SMA treatments. The study lasts about 2 years, wit…

This study is testing a special fabric shoulder brace that mimics muscle action to help people with neuromuscular disorders (like muscular dystrophy or ALS) move their arms better. About 30 participants will be evaluated with and without the brace to see if it improves reach, str…

This study aims to improve genetic counseling for spinal muscular atrophy (SMA) by using advanced DNA analysis to detect a type of gene duplication that current tests miss. About 27 adults who are known carriers or have specific gene patterns will provide samples. The goal is to …

This study looks at the experiences of 200 adults with spinal muscular atrophy (SMA) to understand why some are not receiving approved treatments. Researchers will survey patients about barriers like cost, side effects, or access issues. The goal is to identify gaps in care and h…

This study follows about 175 people with spinal muscular atrophy (SMA) who already received the gene therapy OAV101 in a previous clinical trial. Researchers will monitor them for 5 years to check for any serious side effects and to see how their motor skills develop. The goal is…

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 track walking speed during a 6-minute test and use a wearable sensor to monitor daily activity f…

This study tests a portable digital device that analyzes walking without markers, comparing it to standard motion-capture systems. It includes 30 adults (ages 18-65) with certain neuromuscular diseases or healthy volunteers. The goal is to see if this simpler tool can reliably me…

This study follows up to 90 babies (60 with SMA and 30 healthy) from birth to age 4 to see how their muscles develop. Using special wearable devices worn at home, researchers aim to spot when movement first differs from normal. This will help doctors decide the best time to give …

This study follows 100 children with Duchenne muscular dystrophy or spinal muscular atrophy, identified through newborn screening, to see how their motor skills develop over time. Children wear special sensor garments or ankle monitors at home to track movement. The goal is to fi…