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  • The FDA and Gene Therapy fo...
    Rind, David M

    JAMA : the journal of the American Medical Association, 05/2024, Letnik: 331, Številka: 20
    Journal Article

    This Viewpoint examines the appropriateness of FDA accelerated approval of novel gene therapies to treat boys with Duchenne muscular dystrophy following clinical trials with surrogate outcomes that did not demonstrate net benefits.