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

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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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Wiley 2022-12-01
Series:Pharmacology Research & Perspectives
Subjects:
cystic fibrosis
elexacaftor/tezacaftor/ivacaftor
modulators
sputum
Online Access:https://doi.org/10.1002/prp2.1015

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