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- [45] FDA approves first gene therapy for Duchenne muscular dystrophy, despite internal objections Nature Reviews Drug Discovery, 2023, 22 : 610 - 610
- [48] Revolutionary breakthrough: FDA approves Vyjuvek, the first topical gene therapy for dystrophic epidermolysis bullosa ANNALS OF MEDICINE AND SURGERY, 2023, 85 (12): : 6298 - 6301
- [49] Lessons Learned from the Development of the First FDA-Approved Gene Therapy Drug, Voretigene Neparvovec-rzyl COLD SPRING HARBOR PERSPECTIVES IN MEDICINE, 2023, 13 (05):
- [50] Revolutionary breakthrough: FDA approves CASGEVY, the first CRISPR/Cas9 gene therapy for sickle cell disease ANNALS OF MEDICINE AND SURGERY, 2024, 86 (08): : 4555 - 4559