Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials.

doi:10.1016/S2213-2600(22)00504-5

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Article

Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials.

The Lancet. Respiratory medicine - 1 Jun 2023

Uluer Ahmet Z, MacGregor Gordon, Azevedo Pilar, Indihar Veronica, Keating Claire, Mall Marcus A, McKone Edward F, Ramsey Bonnie W, Rowe Steven M, Rubenstein Ronald C, Taylor-Cousar Jennifer L, Tullis Elizabeth, Yonker Lael M, Chu Chenghao, Lam Anna P, Nair Nitin, Sosnay Patrick R, Tian Simon, Van Goor Fredrick, Viswanathan Lakshmi, Waltz David, Wang Linda T, Xi Yingmei, Billings Joanne, Horsley Alexander

Abstract excerpt

BACKGROUND: Elexacaftor-tezacaftor-ivacaftor has been shown to be safe and efficacious in people with cystic fibrosis and at least one F508del allele. Our aim was to identify a novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination capable of further increasing CFTR-mediated chloride transport, with the potential for once-daily dosing. METHODS: We conducted two phase 2 clinical...

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PMID: 36842446
DOI: 10.1016/S2213-2600(22)00504-5

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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