Comparison of Organoid Swelling and In Vivo Biomarkers of CFTR Function to Determine Effects of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for the F508del Mutation

Am J Respir Crit Care Med. 2020 Dec 1;202(11):1589-1592. doi: 10.1164/rccm.202004-1200LE.
No abstract available

Publication types

  • Comparative Study
  • Letter
  • Observational Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aminophenols / pharmacology
  • Aminophenols / therapeutic use*
  • Aminopyridines / pharmacology
  • Aminopyridines / therapeutic use*
  • Benzodioxoles / pharmacology
  • Benzodioxoles / therapeutic use*
  • Child
  • Chlorides / analysis
  • Colforsin
  • Cystic Fibrosis / drug therapy*
  • Cystic Fibrosis / genetics
  • Cystic Fibrosis / physiopathology
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics
  • Drug Combinations
  • Edema
  • Female
  • Forced Expiratory Volume
  • Homozygote
  • Humans
  • In Vitro Techniques
  • Male
  • Membrane Potentials / physiology
  • Middle Aged
  • Nasal Mucosa / physiopathology
  • Organoids / drug effects*
  • Precision Medicine
  • Prospective Studies
  • Quinolones / pharmacology
  • Quinolones / therapeutic use*
  • Rectum / drug effects*
  • Sweat / chemistry
  • Treatment Outcome
  • Vital Capacity
  • Young Adult

Substances

  • Aminophenols
  • Aminopyridines
  • Benzodioxoles
  • CFTR protein, human
  • Chlorides
  • Drug Combinations
  • Quinolones
  • lumacaftor, ivacaftor drug combination
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • Colforsin