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Article

[Real-world effectiveness of ivacaftor in children with cystic fibrosis and the G551D mutation].

Anales de pediatria - 1 Mar 2019

Gomez-Pastrana David, Nwokoro Chinedu, McLean Mike, Brown Sarah, Christiansen Nanna, Pao Caroline S

Abstract excerpt

INTRODUCTION: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has been shown to improve the nutritional status and lung function of cystic fibrosis patients with the G551D mutation in clinical trials. The objective of this study was to describe the real-world progress of children receiving ivacaftor. METHODS: We describe the real-world progress of four children with...

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Source

Identifiers

PMID: 30093322
DOI: 10.1016/j.anpedi.2018.05.022

Genes

CFTR

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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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[Real-world effectiveness of ivacaftor in children with cystic fibrosis and the G551D mutation].PMID 30093322Read ↗

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