No abstract available
Plain language summary
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.
Publication types
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Research Support, Non-U.S. Gov't
MeSH terms
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Drug Approval
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Dystrophin / genetics
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Genetic Therapy*
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Humans
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Muscular Dystrophy, Duchenne* / genetics
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Muscular Dystrophy, Duchenne* / therapy
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United States
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United States Food and Drug Administration*