Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study.

doi:10.1016/S2213-2600(20)30510-5

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Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study.

The Lancet. Respiratory medicine - 1 Jul 2021

Flume Patrick A, Biner Reta Fischer, Downey Damian G, Brown Cynthia, Jain Manu, Fischer Rainald, De Boeck Kris, Sawicki Gregory S, Chang Philip, Paz-Diaz Hildegarde, Rubin Jaime L, Yang Yoojung, Hu Xingdi, Pasta David J, Millar Stefanie J, Campbell Daniel, Wang Xin, Ahluwalia Neil, Owen Caroline A, Wainwright Claire E

Abstract excerpt

BACKGROUND: Tezacaftor-ivacaftor is an approved cystic fibrosis transmembrane conductance regulator (CFTR) modulator shown to be efficacious and generally safe and well tolerated over 8-24 weeks in phase 3 clinical studies in participants aged 12 years or older with cystic fibrosis homozygous for the Phe508del CFTR mutation (F/F; study 661-106 [EVOLVE]) or heterozygous for the Phe508del CFTR mutation and a...

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PMID: 33581080
DOI: 10.1016/S2213-2600(20)30510-5

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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