Real-world safety and effectiveness of gene therapy for spinal muscular atrophy
Part of Brain & nervous system clinical trials.
This study tracks how well the gene therapy ITVISMA works in everyday medical practice for people with spinal muscular atrophy (SMA). It aims to see if the treatment is safe and effective in a real-world setting.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed genetic diagnosis of spinal muscular atrophy (SMA).
- You are at least 2 years old on the day of the treatment injection.
- Your doctor plans to give you a single dose of ITVISMA gene therapy as part of your routine care.
- You can walk on your own (if under 18) or, if an adult, you either walk on your own or are non-ambulatory but have some arm function.
- Your medical records from the past year are available for review.
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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