Effect of VX-770 in Persons with Cystic Fibrosis and the G551D- <i>CFTR</i> Mutation

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

doi:10.1056/nejmoa0909825

Public

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D- CFTR Mutation

Published in New England Journal of Medicine • Nov 17, 2010

Authors:

Frank J. Accurso

Steven M. Rowe

John P. Clancy

Abstract

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: We ...

Subject

Cystic fibrosis

Medicine

Placebo

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