Risdiplam treatment study for type I and II spinal muscle atrophy
Part of Brain & nervous system clinical trials.
This study follows children with spinal muscular atrophy (SMA)—a genetic condition causing muscle weakness—who are being treated with a medicine called risdiplam. Researchers want to understand how well the treatment works and how it affects daily life in young patients.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child must be diagnosed with type I SMA before age 6 months, or type II SMA before age 5 years
- Treatment with risdiplam must have started within 6 months of diagnosis
- Child must be eating and drinking adequately at study start
- Child must have fully recovered from any recent illness before study start
- Child cannot be on other SMA medicines or enrolled in other drug studies
- Child cannot need a breathing tube or have serious heart, kidney, liver, or digestive problems
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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