New England Journal of Medicine· 2017Q1· Clinical trial
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
- 2,358citations
- Q1SCImago
- 2017year
Short summary
A single intravenous gene therapy infusion significantly improved survival and motor function in infants with Spinal Muscular Atrophy Type 1 (SMA1), with all treated patients alive and event-free at 20 months compared to 8% survival in historical cohorts.
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GeneticsMedicine