FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

Nucleic Acid Ther. 2017 Feb;27(1):1-3. doi: 10.1089/nat.2016.0657. Epub 2016 Dec 8.
No abstract available

MeSH terms

  • Adolescent
  • Child
  • Clinical Trials as Topic
  • Dose-Response Relationship, Drug
  • Drug Approval*
  • Dystrophin / genetics
  • Exons
  • Humans
  • Morpholinos / therapeutic use*
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Muscular Dystrophy, Duchenne / economics
  • Oligonucleotides / therapeutic use
  • Time Factors
  • Treatment Outcome
  • United States
  • United States Food and Drug Administration
  • Walk Test
  • Young Adult

Substances

  • Dystrophin
  • Morpholinos
  • Oligonucleotides
  • PRO051
  • eteplirsen