Elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis harbouring two CFTR Class I variants: real-world data from the French compassionate programmeResearch in context

Summary: Background: The European Medicines Agency has recently expanded the label of elexacaftor-tezacaftor-ivacaftor (ETI) to all people with cystic fibrosis (pwCF) aged 2 years and older who have at least one non-Class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)...

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Bibliographic Details

Published in:	EClinicalMedicine
Main Authors:	Pierre-Régis Burgel, Emmanuelle Girodon, Neeraj Sharma, Caroline Raynal, Jennifer Da Silva, Souphatta Sasorith, Clémence Martin, Isabelle Sermet-Gaudelus, Karen Raraigh
Format:	Article
Language:	English
Published:	Elsevier 2025-10-01
Subjects:	Elexacaftor-tezacaftor-ivacaftor Stop codon Cystic fibrosis CFTR modulators CFTR
Online Access:	http://www.sciencedirect.com/science/article/pii/S2589537025004092