Study of SMA in Children on SMN-Boosting Therapies
Treatments studied
Part of Brain & nervous system clinical trials.
This study is looking for children and teens with spinal muscular atrophy (SMA) who are already on a treatment like Spinraza, Evrysdi, or Zolgensma. The goal is to understand different forms of SMA and how the treatment affects them over time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You or your child must have a confirmed genetic diagnosis of spinal muscular atrophy (SMA).
- You must be currently taking a therapy that boosts the SMN protein, such as Spinraza, Evrysdi, or Zolgensma.
- You must be between 0 and 15 years old (inclusive).
- You cannot be pregnant or breastfeeding (if applicable).
- There must be no other neurological disease or serious condition that could affect the study tests.
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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