Ontology highlight
ABSTRACT:
SUBMITTER: Accurso FJ
PROVIDER: S-EPMC3148255 | biostudies-literature | 2010 Nov
REPOSITORIES: biostudies-literature
Accurso Frank J FJ Rowe Steven M SM Clancy J P JP Boyle Michael P MP Dunitz Jordan M JM Durie Peter R PR Sagel Scott D SD Hornick Douglas B DB Konstan Michael W MW Donaldson Scott H SH Moss Richard B RB Pilewski Joseph M JM Rubenstein Ronald C RC Uluer Ahmet Z AZ Aitken Moira L ML Freedman Steven D SD Rose Lynn M LM Mayer-Hamblett Nicole N Dong Qunming Q Zha Jiuhong J Stone Anne J AJ Olson Eric R ER Ordoñez Claudia L CL Campbell Preston W PW Ashlock Melissa A MA Ramsey Bonnie W BW
The New England journal of medicine 20101101 21
<h4>Background</h4>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.<h4>Methods</h4>We randomly assigned 39 adults with cystic fibrosis and at least one G551D-CFTR allele to receive oral VX-770 every 12 hours at a dose of 25, 75, or 150 mg or placebo for 14 days (in part 1 ...[more]