Can a second drug boost gene therapy in babies with SMA?
NCT ID NCT07444450
First seen Mar 20, 2026 · Last updated May 15, 2026 · Updated 3 times
Summary
This study tests a drug called salanersen in babies with spinal muscular atrophy (SMA) who already received gene therapy. The goal is to see if adding salanersen can improve motor skills and reduce symptoms. About 42 infants with two copies of the SMN2 gene will receive either salanersen or a sham procedure, and all will get salanersen after one year. The study lasts up to 5.5 years and focuses on safety and how well babies develop movement milestones.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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