DUCHENNE MUSCULAR DYSTROPHY

This study is testing whether an oral drug called TAS-205 can help slow the progression of Duchenne muscular dystrophy (DMD). It involves about 104 boys and young men with DMD, split into groups based on whether they can still walk. The main goal is to see if the drug improves th…

This study is testing whether a daily oral medication called EDG-5506 is safe and can help control muscle damage in children and teenagers with Duchenne muscular dystrophy who have already received gene therapy. About 43 participants aged 6-17 will take either the medication or a…

This early-stage study is testing the safety and initial effects of a single intravenous gene therapy called JWK007 in three young boys with Duchenne muscular dystrophy (DMD). The therapy aims to deliver a working copy of a crucial muscle gene that these boys are missing. Researc…

This study is testing an oral medication called EDG-5506 (sevasemten) in children with Duchenne muscular dystrophy (DMD), a serious muscle-wasting condition. Researchers want to see if the drug is safe, how it moves through the body, and if it can reduce signs of muscle damage. T…

This study follows 500 boys and young men with Duchenne muscular dystrophy to compare a new gene therapy (delandistrogene moxeparvovec) against standard steroid treatment in real-world medical settings. Researchers will track walking ability, muscle function, heart health, and sa…

This study follows boys with Duchenne muscular dystrophy who previously received the experimental drug WVE-N531 to see how safe and effective it is over two years. All 175 participants will continue receiving monthly intravenous doses of the drug while researchers monitor their h…

This early-stage study is testing a single dose of an experimental gene therapy called SGT-001 in boys with Duchenne muscular dystrophy (DMD). The main goal is to see if the treatment is safe and well-tolerated. Researchers will also check if the therapy helps produce a needed mu…

This study is testing whether a one-time gene therapy is safe and can help boys with Duchenne muscular dystrophy (DMD) maintain their ability to walk and move. About 99 boys, aged 4 to 7, will receive either the gene therapy or a placebo infusion. The main goal is to see if the t…

This study is tracking the long-term safety and effectiveness of a gene therapy called delandistrogene moxeparvovec (SRP-9001) in people with Duchenne muscular dystrophy. It will follow 400 participants who have already received the therapy in a previous clinical trial. The goal …

This study is tracking boys for up to five years after they received an experimental gene therapy called RGX-202 for Duchenne muscular dystrophy (DMD). The main goal is to understand the long-term safety of the treatment and see if its benefits on muscle strength and movement las…

This study continues testing an experimental treatment called DS-5141b for Duchenne muscular dystrophy. It follows 8 patients who completed an earlier trial to see if long-term use is safe and helps maintain muscle function. Researchers will measure walking ability, muscle streng…

This study is monitoring the long-term safety and effectiveness of the drug vamorolone in boys with Duchenne muscular dystrophy (DMD). It includes 80 boys who have already been taking vamorolone through special access programs. The main goal is to check for side effects like bone…

This study is testing the safety and biological activity of an experimental gene therapy in very young children with Duchenne muscular dystrophy (DMD). It involves 13 children under 4 years old who will receive a single dose of the therapy and be followed for about 5 years. The m…

This study is a long-term follow-up for patients who completed a previous trial for Duchenne muscular dystrophy. It aims to provide continued access to an experimental cell therapy called deramiocel and gather more data on its long-term safety and whether it helps slow the loss o…

This study aims to collect long-term information on how well three specific Duchenne muscular dystrophy (DMD) treatments work in everyday clinical practice. It will follow about 300 boys and young men who are already prescribed these therapies by their doctors. Researchers will t…

This study aims to understand how Duchenne muscular dystrophy affects children's thinking skills and their ability to perform daily activities. Researchers will compare 38 children with DMD to healthy children, measuring their memory, planning, and task performance. The goal is t…

This study aimed to understand how Duchenne muscular dystrophy affects the heart over two years. Researchers planned to use detailed heart MRI scans and blood tests to track changes in heart muscle and function in boys and men with DMD. The study was withdrawn before any particip…