The FDA approval of delandistrogene moxeparvovec-rokl for Duchenne muscular dystrophy: a critical examination of the evidence and regulatory process

Expert Opin Biol Ther. 2024 Sep;24(9):869-871. doi: 10.1080/14712598.2024.2394477. Epub 2024 Aug 20.
No abstract available

Keywords: Duchenne muscular dystrophy; FDA; gene therapy; regulatory.

Publication types

  • Editorial

MeSH terms

  • Drug Approval*
  • Genetic Therapy
  • Humans
  • Muscular Dystrophy, Duchenne* / drug therapy
  • Muscular Dystrophy, Duchenne* / genetics
  • United States
  • United States Food and Drug Administration*