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

Ramsey, B.; Davies, J.; G. Mcelvaney, N.; Tullis, Elizabeth; C Bell, Scott; Drevinek, Pavel; Griese, Matthias; McKone, E.; E. Wainwright, C.; Konstan, M.; Moss, R.; Ratjen, F.; Sermet-Gaudelus, I.; M. Rowe, S.; Dong, Q.; Rodriguez, S.; Yen, Karl; Ordoñez, C.; S. Elborn, J.

doi:10.1056/NEJMoa1105185

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

Shared by NobleBlocks on Nov 3, 2011 • 12:00 AM UTC

Authors:

B. Ramsey

J. Davies

N. G. Mcelvaney

Abstract

BACKGROUND Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis. METHODS We conducted a randomized, double-blind, placebo-controlled trial to evaluate ivacaftor (VX-770), a CFTR potentiator, in subjects 1...

Subject

Medicine

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