期刊
CLINICAL PHARMACOLOGY & THERAPEUTICS
卷 95, 期 6, 页码 592-597出版社
WILEY
DOI: 10.1038/clpt.2014.54
关键词
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资金
- National Institutes of Health (NIH) [U19 GM61390, U01 GM092666, U01 HL0105918, NHLBI-FOA-HL12-035, R24 GM061374]
- Cystic Fibrosis Foundation [R457-CR11, AMIN09YO]
Cystic fibrosis (CF) is a life-shortening disease arising as a consequence of mutations within the CFTR gene. Novel therapeutics for CF are emerging that target CF transmembrane conductance regulator protein (CFTR) defects resulting from specific CFTR variants. Ivacaftor is a drug that potentiates CFTR gating function and is specifically indicated for CF patients with a particular CFTR variant, G551D-CFTR (rs75527207). Here, we provide therapeutic recommendations for ivacaftor based on preemptive CFTR genotype results.
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