Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy: in a clinical trial, all 15 patients were alive and event-free at 20 months of age, as compared with a rate of survival of 8% in a historical cohort.

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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy: in a clinical trial, all 15 patients were alive and event-free at 20 months of age, as compared with a rate of survival of 8% in a historical cohort.