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Article

Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: Safety and efficacy in an expanded access program in the United States.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jan 2016

Taylor-Cousar Jennifer, Niknian Minoo, Gilmartin Geoffrey, Pilewski Joseph M

Abstract excerpt

BACKGROUND: Ivacaftor is the first therapeutic agent approved for the treatment of cystic fibrosis (CF) that targets the underlying molecular defect. Patients with severe lung disease were excluded from the randomized Phase 3 trials. This open-label study was designed to provide ivacaftor to patients in critical medical need prior to commercial product availability. METHODS: CF patients aged ≥6 years with a...

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Source

Identifiers

PMID: 25682022
DOI: 10.1016/j.jcf.2015.01.008

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: Safety and efficacy in an expanded access program in the United States.PMID 25682022Read ↗

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