Real‐life efficacy and safety of elexacaftor/tezacaftor/ivacaftor on severe cystic fibrosis lung disease patients

Abstract Elexacaftor/tezacaftor/ivacaftor (ETI) is a cystic fibrosis (CF) transmembrane conductance regulator modulator, which has shown efficacy in CF patients (≥6 years) with ≥1 Phe508del mutation and a minimal function mutation. In October 2019, ETI became available on compassionate use basis for...

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Bibliografiska uppgifter
Huvudupphovsmän: Renate Kos, Anne H. Neerincx, Dominic W. Fenn, Paul Brinkman, Rianne Lub, Steffie E. M. Vonk, Jolt Roukema, Monique H. Reijers, Suzanne W. J. Terheggen‐Lagro, Josje Altenburg, Christof J. Majoor, Lieuwe D. Bos, Eric G. Haarman, Anke H. Maitland‐van der Zee, the Amsterdam Mucociliary Clearance Disease (AMCD) Research Group
Materialtyp: Artikel
Språk:English
Publicerad: Wiley 2022-12-01
Serie:Pharmacology Research & Perspectives
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Länkar:https://doi.org/10.1002/prp2.1015