‘It’s a vote for hope’: first gene therapy for muscular dystrophy nears approval, but will it work?

Sara Reardon

Nature, 2023, vol. 618, issue 7965, 451-453

Abstract: The FDA’s decision, expected this month, follows several setbacks and delays and will pose difficult choices for the families of children with Duchenne muscular dystrophy.

Keywords: Drug discovery; Gene therapy; Biotechnology (search for similar items in EconPapers)
Date: 2023
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DOI: 10.1038/d41586-023-01799-z

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