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
Elexacaftor/Tezacaftor/Ivacaftor 用于囊性纤维化和罕见 CFTR 变异：体外研究向 3 期双盲、随机、安慰剂对照试验和真实世界研究的转化
期刊:American Journal of Respiratory and Critical Care Medicine
影响因子:19.4
doi:10.1093/ajrccm/aamaf001
Castellani, Carlo; Mondejar-Lopez, Pedro; Van Goor, Fredrick; Quon, Bradley S; Alghisi, Federico; Fabrizzi, Benedetta; Ramsey, Bonnie; Taylor-Cousar, Jennifer L; McKone, Edward F; Tullis, Elizabeth; Weinstock, Tanya G; Tan, Vincent; Ahluwalia, Neil; Mahic, Milada; Liu, Lingyun; Saayman, Sheena; Altshuler, David; Waltz, David; Mall, Marcus A; Fajac, Isabelle
