Duchenne muscular dystrophy

This study tests whether adding sirolimus (a drug that calms the immune system) to ELEVIDYS gene therapy can prevent serious liver injury in boys with Duchenne muscular dystrophy. About 20 participants will receive the treatment and be monitored for side effects. The goal is to m…

This trial tests two ways of taking the steroid prednisolone in boys aged 4-10 with Duchenne muscular dystrophy who can still walk. One group takes the drug every other day, while the other takes it for 10 days on, then 10 days off. The goal is to see which schedule better preser…

This phase 2 trial tests an experimental drug called KER-065 in adult and pediatric males with Duchenne Muscular Dystrophy (DMD), a genetic condition that causes progressive muscle weakness. The study includes both those who can walk and those who cannot, and all participants mus…

This study tests two experimental drugs, ENTR-601-44 and ENTR-601-45, in people with Duchenne muscular dystrophy (DMD) who have already taken part in earlier studies. The goal is to see if long-term use is safe and tolerable, and whether it can help maintain muscle function. Part…

This study tests whether semaglutide (a GLP-1 drug used for weight loss) is safe for adolescents and young adults with Duchenne muscular dystrophy (DMD) who are also overweight or obese. The main concern is that the drug might reduce muscle mass, which is already weak in DMD. Par…

This study tests the safety of a drug called rituximab in 5 boys aged 6-10 with Duchenne muscular dystrophy (DMD) who can still walk. The drug aims to lower certain immune proteins to possibly slow muscle damage. Researchers will monitor side effects and measure changes in walkin…

This study will test different types of strengthening exercises in 45 children aged 6 to 10 with Duchenne muscular dystrophy (DMD). The goal is to see if these exercises can improve muscle strength, walking distance, and chemical markers in the blood. The results could help guide…

This study tests whether a gamified mobile app can help 46 boys aged 6–12 with Duchenne muscular dystrophy stay motivated with their home physiotherapy. The app includes rewards, levels, and feedback to make exercise more engaging. Researchers will measure physical function, endu…

This study tests whether a multicomponent support group can improve the confidence (self-efficacy) of primary caregivers of children with Duchenne muscular dystrophy or spinal muscular atrophy in Pakistan. Thirty caregivers will join group sessions with doctors, therapists, and o…

This study looks at how the absence of a protein called Dp140 in people with Becker muscular dystrophy might affect the brain, thinking, and muscle function. Researchers will use brain and muscle imaging, along with cognitive and psychological tests, to explore these connections.…

This study tests whether a wearable sensor (Syde device) can reliably measure arm and hand function in boys with Duchenne muscular dystrophy who can no longer walk. Current tests are done in clinics and may miss small changes. The goal is to see if home-based digital monitoring i…

This study will follow 500 boys aged 1 to 18 with Duchenne or Becker muscular dystrophy for several years. Researchers will track their muscle function, body composition, lab tests, and heart and lung imaging to map how the disease changes over time. The goal is to create a model…

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 …

This study aims to identify the many factors that influence how physically active children with Duchenne Muscular Dystrophy (DMD) are. Researchers will look at muscle strength, balance, thinking skills, sleep, behavior, and family environment. The goal is to gather information th…