Remote motor development study for early-diagnosed DMD or SMA
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study remotely tracks motor development in children with Duchenne muscular dystrophy or spinal muscular atrophy who were diagnosed at birth or before symptoms started. It aims to see if early monitoring helps improve long-term care and outcomes.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have a confirmed diagnosis of either Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA).
- For SMA, they need to have exactly 4 copies of the SMN2 gene (or be eligible for monitoring).
- Your child should have been diagnosed through newborn screening, family testing, or before symptoms appeared.
- For DMD, your child must be younger than 4 years old at the time of joining.
- A parent or legal guardian must be able to give permission for your child to participate.
- Your family needs to have internet access at home for the study's check-ins.
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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