New study to track gene Therapy's Real-World impact on spinal muscular atrophy
NCT ID NCT07448610
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study will follow 36 people with spinal muscular atrophy (SMA) who receive a gene therapy called onasemnogene abeparvovec (ITVISMA) injected into the spinal fluid. The goal is to see how well it works and how safe it is in everyday medical practice. Participants must be at least 2 years old and have a confirmed SMA diagnosis.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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Other studies related to the condition(s) this trial covers.
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