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- W4385973071 abstract "The path to effective therapies across the landscape of the approximately 7,000 rare genetic diseases has been both tortuous and transformative. The Duchenne muscular dystrophy (DMD) experience represents the former and may be emblematic of the challenges faced when developing therapies for those genetic diseases with high unmet need but variable presentations that ultimately progress to disabling and fatal outcomes. To date, the FDA has approved four treatments in DMD, all through the mechanism of “accelerated approval” (AA). However, to date, none of those four treatments have received full FDA approval. These and other complexities were visible at a May 2023 meeting of the FDA’s Cellular and Tissue Gene Therapies Advisory Committee (CTGTAC) where the first request for AA for a gene therapy (GT) delivered by an adeno-associated viral (AAV) vector was considered." @default.
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- W4385973071 date "2023-09-01" @default.
- W4385973071 modified "2023-10-06" @default.
- W4385973071 title "Challenges in accelerated approvals for gene therapies" @default.
- W4385973071 cites W3138386431 @default.
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- W4385973071 doi "https://doi.org/10.1016/j.ymthe.2023.08.004" @default.
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