[HTML][HTML] A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation

…, F Ratjen, I Sermet-Gaudelus, SM Rowe… - … England Journal of …, 2011 - Mass Medical Soc
Background Increasing the activity of defective cystic fibrosis transmembrane conductance
regulator (CFTR) protein is a potential treatment for cystic fibrosis. Methods We conducted a …

Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial

…, PA Flume, JS Elborn, J Cooke, SM Rowe… - The Lancet …, 2015 - thelancet.com
Background Ivacaftor has been previously assessed in patients with cystic fibrosis with
Gly551Asp-CFTR or other gating mutations. We assessed ivacaftor in patients with Arg117His-…

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double …

…, EF McKone, DG Downey, E Van Braeckel, SM Rowe… - The Lancet, 2019 - thelancet.com
Background Cystic fibrosis transmembrane conductance regulator (CFTR) modulators correct
the basic defect caused by CFTR mutations. Improvements in health outcomes have been …

[HTML][HTML] Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

…, A Munck, F Ratjen, SM Rowe… - … England Journal of …, 2015 - Mass Medical Soc
Background Cystic fibrosis is a life-limiting disease that is caused by defective or deficient
cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is the …

[HTML][HTML] Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with a single Phe508del allele

…, S Tian, D Waltz, F Xuan, SM Rowe… - … England Journal of …, 2019 - Mass Medical Soc
Background Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis
transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at …

A revised airway epithelial hierarchy includes CFTR-expressing ionocytes

…, H Bihler, M Mense, GJ Tearney, SM Rowe… - Nature, 2018 - nature.com
The airways of the lung are the primary sites of disease in asthma and cystic fibrosis. Here
we study the cellular composition and hierarchy of the mouse tracheal epithelium by single-…

[HTML][HTML] Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation

FJ Accurso, SM Rowe, JP Clancy… - … England Journal of …, 2010 - Mass Medical Soc
Background A new approach in the treatment of cystic fibrosis involves improving the function
of mutant cystic fibrosis transmembrane conductance regulator (CFTR). VX-770, a CFTR …

The future of cystic fibrosis care: a global perspective

…, C O'Neill, U Pypops, KS Raraigh, SM Rowe… - The Lancet …, 2020 - thelancet.com
The past six decades have seen remarkable improvements in health outcomes for people
with cystic fibrosis, which was once a fatal disease of infants and young children. However, …

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

JP Clancy, SM Rowe, FJ Accurso, ML Aitken, RS Amin… - Thorax, 2012 - thorax.bmj.com
Background VX-809, a cystic fibrosis transmembrane conductance regulator (CFTR) modulator,
has been shown to increase the cell surface density of functional F508del-CFTR in vitro. …

[HTML][HTML] VX-445–tezacaftor–ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles

…, EF McKone, BW Ramsey, SM Rowe… - New england journal …, 2018 - Mass Medical Soc
Background VX-445 is a next-generation cystic fibrosis transmembrane conductance regulator
(CFTR) corrector designed to restore Phe508del CFTR protein function in patients with …