Long-term study of experimental treatment for duchenne muscular dystrophy
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This long-term study continues to test an experimental treatment for Duchenne muscular dystrophy that works by helping the body skip over faulty parts of the gene. It is only open to people who already finished one of the earlier studies of this drug, to see if it remains safe and effective over a longer time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have completed one of the two earlier studies for this treatment
- You need to be willing to give consent (if adult) or assent (if minor)
- If you are sexually active with a female partner who could become pregnant, you must use condoms during sex
- You should not have any new health problems that would make the study unsafe for you
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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