Nanomolar-potency ‘co-potentiator’ therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants

VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

D. Keating, Gautham Marigowda, Lucy Burr et al. · New England Journal of Medicine · 2018 · 707 citations · Full text

From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations

Radu G. Avramescu, Annette Chiang, Scott A. Houck et al. · Molecular Biology of the Cell · 2016 · 582 citations · Full text

Pathology, Combinatorial Pharmacotherapy, Synaptic Signaling +28

Ivacaftor potentiation of multiple CFTR channels with gating mutations

Haihui Yu, Bill Burton, Chien-Jung Huang et al. · Journal of Cystic Fibrosis · 2012 · 414 citations · Full text

Knockout Mouse, Signal Transduction, Molecular Physiology +7

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial

Eitan Kerem, Michael W. Konstan, K. De Boeck et al. · The Lancet Respiratory Medicine · 2014 · 333 citations · Full text

Allergy, Placebo-controlled Phase 3, Clinical Trials +3

Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function

Fredrick Van Goor, Haihui Yu, Bill Burton et al. · Journal of Cystic Fibrosis · 2013 · 332 citations

Protein Processing, Missense Mutations, Cftr Forms +9