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

Simon Y. Graeber, Peter van Mourik, Annelotte M. Vonk, Evelien Kruisselbrink, Stephanie Hirtz, Cornelis K. van der Ent, Marcus A. Mall*, Jeffrey M. Beekman

*Corresponding author for this work

Research output: Contribution to journalLetterAcademicpeer-review

7 Citations (Scopus)
Original languageEnglish
Pages (from-to)1589-1592
Number of pages4
JournalAmerican Journal of Respiratory and Critical Care Medicine
Volume202
Issue number11
DOIs
Publication statusPublished - 1 Dec 2020

Keywords

  • Adolescent
  • Adult
  • Aminophenols/pharmacology
  • Aminopyridines/pharmacology
  • Benzodioxoles/pharmacology
  • Child
  • Chlorides/analysis
  • Colforsin
  • Cystic Fibrosis Transmembrane Conductance Regulator/genetics
  • Cystic Fibrosis/drug therapy
  • 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
  • Rectum/drug effects
  • Sweat/chemistry
  • Treatment Outcome
  • Vital Capacity
  • Young Adult

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