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

Ivacaftor has been previously assessed in patients with cystic fibrosis with Gly551Asp-CFTR or other gating mutations. We assessed ivacaftor in patients with Arg117His-CFTR, a residual function mutation. We did a 24-week, placebo-controlled, double-blind, randomised clinical trial, which enrolled 69...

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Vydáno v:	The lancet respiratory medicine Ročník 3; číslo 7; s. 524 - 533
Hlavní autoři:	Moss, Richard B, Flume, Patrick A, Elborn, J Stuart, Cooke, Jon, Rowe, Steven M, McColley, Susanna A, Rubenstein, Ronald C, Higgins, Mark
Médium:	Journal Article
Jazyk:	angličtina
Vydáno:	England 01.07.2015
Témata:	Adolescent Adult Aged Aminophenols - therapeutic use Child Chloride Channels - drug effects Cystic Fibrosis - drug therapy Cystic Fibrosis - genetics Cystic Fibrosis Transmembrane Conductance Regulator - genetics Double-Blind Method Female Forced Expiratory Volume - drug effects Humans Male Middle Aged Mutation - genetics Quinolones - therapeutic use Respiratory System Agents - therapeutic use Treatment Outcome Young Adult
ISSN:	2213-2619
On-line přístup:	Zjistit podrobnosti o přístupu
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