Article
Understanding and addressing the needs of people with cystic fibrosis in the era of CFTR modulator therapy.
The Lancet. Respiratory medicine - 1 Oct 2023
Hisert Katherine B, Birket Susan E, Clancy John Paul, Downey Damian G, Engelhardt John F, Fajac Isabelle, Gray Robert D, Lachowicz-Scroggins Marrah E, Mayer-Hamblett Nicole, Thibodeau Patrick, Tuggle Katherine L, Wainwright Claire E, De Boeck Kris
Abstract excerpt
Cystic fibrosis is a multiorgan disease caused by impaired function of the cystic fibrosis transmembrane conductance regulator (CFTR). Since the introduction of the CFTR modulator combination elexacaftor-tezacaftor-ivacaftor (ETI), which acts directly on mutant CFTR to enhance its activity, most people with cystic fibrosis (pwCF) have seen pronounced reductions in symptoms, and studies project marked increases in...
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