Elexacaftor/tezacaftor/ivacaftor for cystic fibrosis and rare CFTR variants: in vitro translation to a phase 3, double-blind, randomized, placebo-controlled trial, and real-world study.
Castellani C, Mondejar-Lopez P, Van Goor F, Quon BS, Alghisi F, Fabrizzi B, Ramsey B, Taylor-Cousar JL, McKone EF, Tullis E, Weinstock TG, Tan V, Ahluwalia N, Mahic M, Liu L, Saayman S, Altshuler D, Waltz D, Mall MA, Fajac I. Elexacaftor/tezacaftor/ivacaftor for cystic fibrosis and rare CFTR variants: in vitro translation to a phase 3, double-blind, randomized, placebo-controlled trial, and real-world study. Am J Respir Crit Care Med. 2026 Feb 01; 212(2):327-337.