Muscle function study in SMA patients on SMN therapies
Part of Brain & nervous system clinical trials.
This study looks at how muscles work in children and young adults with spinal muscular atrophy (SMA) who are taking or starting SMN-directed therapies (like Evrysdi, Spinraza, or Zolgensma). It aims to understand the body's response to these treatments, which could help improve future care.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a genetic test showing SMA with two missing or mutated copies of the SMN1 gene.
- You must have 2, 3, or 4 copies of the SMN2 gene.
- You must be between 5 and 20 years old.
- If you cannot walk, you must be able to sit or stand with support, and have a motor function score (HFMSE) between 10 and 45.
- If you can walk independently, you must be able to walk at least 100 meters without help, and have a motor function score (HFMSE) between 40 and 66.
- You must be on or planning to take one of these SMA medications: Evrysdi, Spinraza, or Zolgensma, or have never taken any of them.
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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