Journal of cystic fibrosis : official journal of the European Cystic Fibrosis SocietyHumansCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorAminophenolsQuinolones
Global prevalence of CFTR variants with respect to their responsiveness to elexacaftor-tezacaftor-ivacaftor.
P-R Burgel, A Orenti, E Cromwell, M Macek, H H Gutierrez, B Karadag, A Faro, J G van Rens, L Naehrlich, E Bakkeheim, S B Carr, A Lindblad, A Zolin, E Lammertyn, R Ruseckaite
Published: 202510.1016/j.jcf.2025.10.007
Abstract
BACKGROUND: Elexacaftor-tezacaftor-ivacaftor (ETI) is mostly approved in people with cystic fibrosis (pwCF) with a p.Phe508del CFTR variant. The US Food and Drug Administration (FDA) approved ETI for an additional 177 rare CFTR variants and studies i…
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