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: We randomly assigned 39 adults with cystic fibrosis and at least one G551D-CFTR allele to receive oral VX-770 every 12...
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