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Cystic fibrosis airway inflammation enables elexacaftor/tezacaftor/ivacaftor-mediated rescue of N1303KCFTRmutation

9 Nov 2023

Abstract excerpt

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators promote robust clinical improvements in people with cystic fibrosis (pwCF) with qualifying genotypes [1, 2]. A triple combination therapy consisting of two CFTR folding correctors (elexacaftor and tezacaftor) plus CFTR potentiator ivacaftor, referred to as ETI, has been approved by the FDA and European agencies ( , ; ) as highly effective...

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PMID: 38226069
DOI: 10.1183/23120541.00746-2023

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