A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F11%3A7160" target="_blank" >RIV/00064203:_____/11:7160 - isvavai.cz</a>
Alternative codes found
RIV/00216208:11130/11:7160
Result on the web
<a href="http://www.ncbi.nlm.nih.gov/pubmed/22047557" target="_blank" >http://www.ncbi.nlm.nih.gov/pubmed/22047557</a>
DOI - Digital Object Identifier
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Alternative languages
Result language
angličtina
Original language name
A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation
Original language description
Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis. We conducted a randomized, double-blind, placebo-controlled trial to evaluate ivacaftor (VX-770), a CFTRpotentiator, in subjects 12 years of age or older with cystic fibrosis and at least one G551D-CFTR mutation. Subjects were randomly assigned to receive 150 mg of ivacaftor every 12 hours (84 subjects, of whom 83 received at least one dose) or placebo (83, of whom 78 received at least one dose) for 48 weeks. The primary end point was the estimated mean change from baseline through week 24 in the percent of predicted forced expiratory volume in 1 second (FEV(1)). The change from baseline through week 24in the percent of predicted FEV1 was greater by 10.6 percentage points in the ivacaftor group than in the placebo group (P < 0.001). Effects on pulmonary function were noted by 2 weeks, and a significant treatment effect was maintained th
Czech name
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Czech description
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Classification
Type
J<sub>x</sub> - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)
CEP classification
FP - Other medical fields
OECD FORD branch
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Result continuities
Project
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Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2011
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
New England Journal of Medicine
ISSN
0028-4793
e-ISSN
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Volume of the periodical
365
Issue of the periodical within the volume
18
Country of publishing house
US - UNITED STATES
Number of pages
10
Pages from-to
1663-1672
UT code for WoS article
000296424200005
EID of the result in the Scopus database
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