Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

Abstract

Elexacaftor-tezacaftor-ivacaftor was efficacious in patients with cystic fibrosis with Phe508del-minimal function genotypes, in whom previous CFTR modulator regimens were ineffective. (Funded by Vertex Pharmaceuticals; VX17-445-102 ClinicalTrials.gov number, NCT03525444.).

Keywords

Affiliated Institutions

• Seattle Children's Hospital US
• KU Leuven BE
• Westmead Institute for Medical Research AU
• University College Dublin IE
• St. Michael's Hospital CA
• University of Kansas Medical Center US
• St. Vincent's University Hospital IE
• Vertex Pharmaceuticals (United States) US
• Charles University CZ
• University of Alabama at Birmingham US
• University of Colorado Denver US
• McGill University Health Centre CA
• University of Toronto CA
• The University of Texas Southwestern Medical Center US
• The University of Sydney AU
• Westmead Hospital AU
• University of Washington US
• National Jewish Health US
• German Center for Lung Research DE

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