Ataluren, a New Therapeutic for Alpha-1 Antitrypsin-Deficient Individuals with Nonsense Mutations

Am J Respir Crit Care Med. 2018 Oct 15;198(8):1099-1102. doi: 10.1164/rccm.201802-0338LE.
No abstract available

Publication types

  • Letter
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Codon, Nonsense / genetics*
  • Humans
  • Middle Aged
  • Oxadiazoles / therapeutic use*
  • alpha 1-Antitrypsin / metabolism
  • alpha 1-Antitrypsin Deficiency / drug therapy*
  • alpha 1-Antitrypsin Deficiency / genetics

Substances

  • Codon, Nonsense
  • Oxadiazoles
  • alpha 1-Antitrypsin
  • ataluren