Once-weekly steroid for very young children with DMD
Treatments studied
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests whether a steroid taken once each week is safe and useful for children with Duchenne muscular dystrophy (DMD). It aims to help slow disease effects in very young children whose diagnosis and lab tests confirm DMD.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child is between 1 month and 30 months old
- A clinician sees weakness that fits Duchenne muscular dystrophy (DMD) on exam
- Blood test shows creatine kinase at least 20 times the usual upper limit
- Your child has a genetic test showing a mutation known to cause DMD
- Your child has NOT already taken corticosteroids (steroid medicines) before
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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