Ronald C. Rubenstein

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 · 1.3K citations · Full text

Mendelian Disorder, Genetics, Ivacaftor Combination Regimen +2

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D- <i>CFTR</i> Mutation

Frank J. Accurso, Steven M. Rowe, John P. Clancy et al. · New England Journal of Medicine · 2010 · 798 citations · Full text

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the <i>F508del-CFTR</i> mutation

John P. Clancy, Steven M. Rowe, Frank J. Accurso et al. · Thorax · 2011 · 493 citations · Full text

In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR.

Ronald C. Rubenstein, Marie E. Egan, Pamela L. Zeitlin · Journal of Clinical Investigation · 1997 · 385 citations · Full text

Molecular Regulation, Cellular Physiology, Molecular Pharmacology +21

Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR : a randomised, placebo-controlled phase 3 trial

Félix Ratjen, Christopher Hug, Gautham Marigowda et al. · The Lancet Respiratory Medicine · 2017 · 291 citations

Pulmonary Fibrosis, Medicine, Placebo-controlled Phase 3