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AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: end-of-Study event free survival and achievement of developmental milestones

Gene TherapiesTranslational MedicineDevelopmental BiologyNovel TherapyIn Vivo Gene TherapyGeneticsClinical TrialsTranslational ResearchAvxs-101 Phase 1Antisense TherapyDisease Gene IdentificationGene VectorDevelopmental MilestonesMedicineSma Type 1Genome Editing