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Article

Clinical and functional efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis carrying the N1303K mutation.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Nov 2023

Sadras Ido, Kerem Eitan, Livnat Galit, Sarouk Ifat, Breuer Oded, Reiter Joel, Gileles-Hillel Alex, Inbar Ori, Cohen Michael, Gamliel Ayelet, Stanleigh Noemie, Gunawardena Tarini, Bartlett Claire, Gonska Tanja, Moraes Theo, Eckford Paul D W, Bear Christine E, Ratjen Felix, Kerem Batsheva, Wilschanski Michael, Shteinberg Michal, Cohen-Cymberknoh Malena

Abstract excerpt

BACKGROUND: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) significantly improves health outcomes in people with cystic fibrosis (pwCF) carrying one or two F508del mutations. According to in vitro assays performed in FRT cells, 178 additional mutations respond to ELX/TEZ/IVA. The N1303K mutation is not included in this list of mutations. Recent in vitro data suggested that ELX/TEZ/IVA increases N1303K-CFTR...

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Aminophenols
Benzodioxoles
Chloride Channel Agonists
Cystic Fibrosis

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Cystic Fibrosis Research Advances

Cystic Fibrosis Transmembrane Conductance Regulator

Neonatal Respiratory Health Research

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PMID: 37331863
DOI: 10.1016/j.jcf.2023.06.001

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Clinical and functional efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis carrying the N1303K mutation.PMID 37331863Read ↗

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