Elexacaftor co-potentiates the activity of F508del and gating mutants of CFTR

A CFTR Potentiator in Patients with Cystic Fibrosis and the <i>G551D</i> Mutation

Bonnie W. Ramsey, Jane C. Davies, Noel G. McElvaney et al. · New England Journal of Medicine · 2011 · 2.2K citations · Full text

Medicine, Molecular Biology, Molecular Genetics +8

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

Peter G. Middleton, Marcus Mall, Pavel Dřevı́nek et al. · New England Journal of Medicine · 2019 · 2.1K citations · Full text

Life Sciences, Pharmacology, Pharmacotherapy +16

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

Harry Heijerman, Edward F. McKone, D.G. Downey et al. · The Lancet · 2019 · 1.4K citations · Full text

Life Sciences, Genetics, Pharmacology +14

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 · 590 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +17

Mechanism-based corrector combination restores ΔF508-CFTR folding and function

Tsukasa Okiyoneda, Guido Veit, Johanna F. Dekkers et al. · Nature Chemical Biology · 2013 · 390 citations · Full text

Life Sciences, Molecular Biology, Molecular Genetics +10