Gene therapy for Duchenne muscular dystrophy
Treatments studied
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study is checking how safe and effective ELEVIDYS, a gene therapy for Duchenne muscular dystrophy, is when used in everyday clinics. It will help doctors understand more about the treatment's real-world effects and provide care for those who receive it.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be male, born male, and able to walk on your own (with or without help).
- You must be at least 4 years old at the time of treatment.
- You must be eligible to receive ELEVIDYS as part of your regular care (for the first group).
- If you are sexually active, both you and your partner must agree to use birth control during the study.
- You or your legal guardian must be willing and able to follow the study schedule and give informed consent.
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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