Double-Duty treatment: new drug aims to boost effects of SMA gene therapy in infants
NCT ID NCT07444450
Summary
This study is testing whether adding a drug called salanersen can improve outcomes for babies with spinal muscular atrophy (SMA) who have already received a one-time gene therapy. Researchers want to see if this follow-up treatment is safe and if it helps babies gain more movement skills. The study will involve about 42 infants who received the gene therapy very early in life, before showing SMA symptoms.
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Conditions
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