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Article

Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis.

Health technology assessment (Winchester, England) - 1 Mar 2014

Whiting Penny, Al Maiwenn, Burgers Laura, Westwood Marie, Ryder Steve, Hoogendoorn Martine, Armstrong Nigel, Allen Alex, Severens Hans, Kleijnen Jos

Abstract excerpt

BACKGROUND: Ivacaftor (Kalydeco(®), Vertex Pharmaceuticals) is the first of a new class of drugs that target the underlying protein defect in cystic fibrosis (CF). It is aimed at patients with the G551D (glycine to aspartate change in nucleotide 1784 in exon 11) mutation; 5.7% of patients with CF...

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PMID: 24656117
DOI: 10.3310/hta18180
PMCID: PMC4780965

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-24656117-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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