MUSCULAR DYSTROPHY

This study is testing the safety of a single-dose gene therapy called AB-1003 in adults with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle disorder. Researchers will give increasing doses to small groups of participants to check for side effects. The goal is to…

This study is testing a brain implant system called BrainGate2 to see if it's safe and if it works. People with paralysis from conditions like spinal cord injury or ALS have a small sensor placed in their brain. The main goal is to see if they can use their thoughts to control a …

This study is testing whether a robotic exoskeleton worn on the legs can improve walking ability in children with movement disorders like cerebral palsy or spina bifida. Children aged 3 to 17 will use the device at home for 12 weeks to see if it helps their knee movement and walk…

This study aims to better understand a wide range of inherited neurological disorders that affect nerves, muscles, and the brain. Researchers will observe up to 3,500 participants, including people with these conditions and their healthy relatives, to track symptoms, disease prog…

This study aims to see if ultrasound can be a better tool for measuring the health of nerves and muscles. Researchers will scan both healthy adults and people with nerve or muscle disorders to collect normal measurements and see how they differ in disease. The goal is to gather i…

This study is testing a portable digital device that measures walking patterns without needing complex lab equipment. Researchers want to see if this simple device can accurately track how people with neuromuscular diseases walk compared to healthy volunteers. The goal is to crea…

This study creates a national registry to connect people with myotonic dystrophy or facioscapulohumeral muscular dystrophy (FSHD) with researchers. It aims to better understand these rare muscle diseases by tracking symptoms over time and helping match interested participants to …

This study aims to find the specific genes that cause inherited heart and muscle diseases like cardiomyopathy and muscular dystrophy. Researchers will analyze DNA from up to 2,000 patients and their family members who have these conditions. The goal is to better understand the ge…