Exon Skipping Therapy

Cell. 2016 Nov 17;167(5):1144. doi: 10.1016/j.cell.2016.10.050.

Abstract

Exondys 51 is the first therapy for Duchenne muscular dystrophy (DMD) to have been granted accelerated approval by the FDA. Approval was granted based on using dystrophin expression as a surrogate marker. Exondys 51 targets DMD exon 51 for skipping to restore the reading frame for 13% of Duchenne patients.

MeSH terms

  • Dystrophin / genetics*
  • Exons
  • Genetic Therapy*
  • Humans
  • Muscular Dystrophy, Duchenne / genetics*
  • Muscular Dystrophy, Duchenne / therapy*
  • Reading Frames
  • United States
  • United States Food and Drug Administration

Substances

  • Dystrophin