Long-term safety study of Zolgensma for spinal muscular atrophy
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study follows the long-term safety of Zolgensma gene therapy in Brazilian children with spinal muscular atrophy (SMA). It helps doctors understand how safe the treatment is over time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of spinal muscular atrophy (SMA) with specific genetic changes.
- You must have already been treated with Zolgensma (gene therapy).
- You cannot be currently taking part in any other interventional clinical trial, except the OFELIA study.
- You and your parent or guardian must be willing to have phone check-ups as the study requires.
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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