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Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation
Author(s) -
Frank J. Accurso,
Steven M. Rowe,
John P. Clancy,
Michael Boyle,
Jordan Dunitz,
Peter R. Durie,
Scott D. Sagel,
Douglas B. Hornick,
Michael W. Konstan,
Scott H. Donaldson,
Richard B. Moss,
Joseph M. Pilewski,
Ronald C. Rubenstein,
Ahmet Uluer,
Moira L. Aitken,
Steven D. Freedman,
Lynn M. Rose,
Nicole Mayer-Hamblett,
Qunming Dong,
Jiuhong Zha,
Anne J. Stone,
Eric R. Olson,
Claudia L. Ordoñez,
Preston W. Campbell,
Melissa A. Ashlock,
Bonnie W. Ramsey
Publication year - 2010
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa0909825
Subject(s) - cystic fibrosis , medicine , placebo , ivacaftor , cystic fibrosis transmembrane conductance regulator , adverse effect , gastroenterology , anesthesia , urology , pathology , alternative medicine
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.

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