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Article

Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Mar 2021

Guimbellot J S, Baines A, Paynter A, Heltshe S L, VanDalfsen J, Jain M, Rowe S M, Sagel S D

Abstract excerpt

BACKGROUND: The cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, ivacaftor, was first approved for people with CF and the G551D CFTR mutation. This study describes the long-term clinical effectiveness of ivacaftor in this population. METHODS: We conducted a multicenter, prospective, longitudinal, observational study of people with CF ages ≥6 years with at least one copy of the G551D CFTR...

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Identifiers

PMID: 33249004
DOI: 10.1016/j.jcf.2020.11.008
PMCID: PMC8183611

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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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