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Article

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

The Lancet. Respiratory medicine - 1 Jul 2015

Moss Richard B, Flume Patrick A, Elborn J Stuart, Cooke Jon, Rowe Steven M, McColley Susanna A, Rubenstein Ronald C, Higgins Mark

Abstract excerpt

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-CFTR, a residual function mutation. METHODS: We did a 24-week, placebo-controlled, double-blind, randomised clinical trial, which enrolled 69 patients with cystic fibrosis aged 6 years and older with Arg117His-CFTR and percentage of...

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Identifiers

PMID: 26070913
DOI: 10.1016/S2213-2600(15)00201-5
PMCID: PMC4641035

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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