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Broad FDA Approval of Gene Therapy Expands Treatment Choices for Duchenne Muscular Dystrophy: Debra Miller & Michael ...
WATCH TIME: 9 minutes "Our job today is far from done in this disease, and we've improved the whole therapeutic landscape of exon skipping. We need to do exactly the same with gene therapy." Originally SRP-9001 (Elevidys; Sarepta Therapeutics …