Exon-skipping therapy: a roadblock, detour, or bump in the road?

Sci Transl Med. 2014 Apr 2;6(230):230fs14. doi: 10.1126/scitranslmed.3008873.

Abstract

Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.

MeSH terms

  • Clinical Trials as Topic
  • Dystrophin / genetics
  • Dystrophin / therapeutic use
  • Exons / genetics*
  • Genetic Therapy*
  • Humans
  • Muscular Dystrophies / genetics
  • Muscular Dystrophies / therapy

Substances

  • Dystrophin