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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Váldodahkkit:	Pierre-Régis Burgel, Emmanuelle Girodon, Neeraj Sharma, Caroline Raynal, Jennifer Da Silva, Souphatta Sasorith, Clémence Martin, Isabelle Sermet-Gaudelus, Karen Raraigh
Materiálatiipa:	Artigo
Giella:	Inglês
Almmustuhtton:	Elsevier 2025-10-01
Ráidu:	EClinicalMedicine
Fáttát:	Elexacaftor-tezacaftor-ivacaftor Stop codon Cystic fibrosis CFTR modulators CFTR
Liŋkkat:	http://www.sciencedirect.com/science/article/pii/S2589537025004092
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