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

W. Ramsey, Bonnie; C. Davies, Jane; G. McElvaney, Noel; Tullis, Elizabeth; C. Bell, Scott; Dřevínek, Pavel; Griese, Matthias; F. McKone, Edward; Wainwright, Claire; W. Konstan, Michael; B. Moss, Richard; Ratjen, Félix; Sermet‐Gaudelus, Isabelle; M. Rowe, Steven; Dong, Qunming; Rodriguez, Sally; Yen, Karl; L. Ordoñez, Claudia; Elborn, J.S.

doi:10.1056/nejmoa1105185

Public

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

Published in New England Journal of Medicine • Nov 2, 2011

Authors:

Bonnie W. Ramsey

Jane C. Davies

Noel 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...

Subject

Potentiator

Cystic fibrosis

Medicine

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