MUSCULAR DYSTROPHY

This study looks at two add-on treatments for Duchenne muscular dystrophy (DMD): a drug called tadalafil that improves blood flow, and aerobic exercise training. The goal is to see if these can improve muscle function and delay disease progression. About 50 ambulatory children wi…

This study tests a single dose of a gene therapy called AB-1003 in 10 adults with limb girdle muscular dystrophy type 2I/R9, a genetic condition that weakens muscles. The main goal is to check safety and find the right dose. Participants must be able to walk 10 meters in under 30…

This study tests whether taking amino acid supplements, along with a structured diet and exercise program, can help people with facioscapulohumeral muscular dystrophy (FSHD) gain muscle, lose fat, and improve physical performance. The trial includes 48 adults aged 18 to 50 with F…

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 a robotic leg brace (exoskeleton) designed to improve walking in children aged 3 to 17 with conditions like cerebral palsy, spina bifida, muscular dystrophy, or incomplete spinal cord injury. The device can assist or resist leg movement during walking. The trial …

This study aims to find the genes that cause inherited heart and muscle diseases, such as cardiomyopathy, arrhythmia, and muscular dystrophy. Researchers will analyze DNA from patients and their families to identify mutations. The goal is to better understand these conditions, no…

This study aims to learn more about rare inherited neurological disorders by observing up to 3,500 people with these conditions and their relatives. Researchers will collect medical history, perform exams, and take blood or skin samples to study genetics and disease progression. …

This study aims to improve how doctors observe nerves and muscles using ultrasound, a safe imaging technique. Researchers will collect normal measurements from healthy adults and compare them to people with neuromuscular disorders. Participants will have up to 5 clinic visits for…

This study tests a brain implant that may allow people with paralysis (from spinal cord injury, ALS, or similar conditions) to control a computer cursor or other devices using just their thoughts. The main goal is to check the device's safety and see if it works as a proof of con…

This study creates a national registry for people with myotonic dystrophy (DM) or facioscapulohumeral muscular dystrophy (FSHD) and their family members. The goal is to connect patients with researchers to speed up understanding of these inherited muscle-weakening diseases. Parti…

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…