Gene therapy for non-ambulatory Duchenne muscular dystrophy
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study tests a gene therapy that aims to help muscles produce a protein they are missing. It is for people with Duchenne muscular dystrophy who are no longer able to walk, and it may help slow the progression of the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of Duchenne muscular dystrophy (DMD).
- You are no longer able to walk (non-ambulatory).
- You have some arm and hand function, as measured by a specific test.
- If you are in certain groups, you must be on a stable dose of steroids for at least 12 weeks before the study.
- You have not had gene therapy or certain other treatments recently.
- You do not have other health conditions that would make gene therapy risky.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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