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Article

Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation.

The New England journal of medicine - 18 Nov 2010

Accurso Frank J, Rowe Steven M, Clancy J P, Boyle Michael P, Dunitz Jordan M, Durie Peter R, Sagel Scott D, Hornick Douglas B, Konstan Michael W, Donaldson Scott H, Moss Richard B, Pilewski Joseph M, Rubenstein Ronald C, Uluer Ahmet Z, Aitken Moira L, Freedman Steven D, Rose Lynn M, Mayer-Hamblett Nicole, Dong Qunming, Zha Jiuhong, Stone Anne J, Olson Eric R, Ordoñez Claudia L, Campbell Preston W, Ashlock Melissa A, Ramsey Bonnie W

Abstract excerpt

BACKGROUND: A new approach in the treatment of cystic fibrosis involves improving the function of mutant cystic fibrosis transmembrane conductance regulator (CFTR). VX-770, a CFTR potentiator, has been shown to increase the activity of wild-type and defective cell-surface CFTR in vitro. METHODS:...

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Identifiers

PMID: 21083385
DOI: 10.1056/NEJMoa0909825
PMCID: PMC3148255

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-21083385-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 2 Sept 2026.

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