Progressive muscular dystrophy

This trial tests an experimental drug called SFL-0821 in adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. Researchers give the drug by intravenous infusion, either once or multiple times, and compare it agains…

This early-stage trial tests amlodipine, a calcium channel blocker used for high blood pressure, in 20 adults with myotonic dystrophy type 1. The goal is to see if the drug is safe and can improve muscle strength, reduce stiffness, and help with daily function. All participants w…

This study follows 100 adults with myotonic dystrophy (types 1 or 2) for two years to understand how muscle stiffness, daily function, and heart health change over time. Researchers will look back at up to 18 months of past medical records and then track participants with clinic …

This study is creating a registry for people with Duchenne and Becker muscular dystrophy, as well as symptomatic female carriers. The goal is to collect health data and quality-of-life information to monitor how new therapies work in real-world settings. Up to 1,500 participants …