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VX-770
Indications:
- treatment of cystic fibrosis (with G551D mutation of CTFR)
Dosage: 150-250 mg PO BID
Adverse effects:
- macular rash
- deterioration of glycemic control in diabetes
Mechanism of action:
- potentiates function of CTFR
General
metabolic agent (metabolic modifier)
References
- Accurso FJ et al.
Effect of VX-770 in persons with cystic fibrosis and the
G551D-CFTR mutation.
N Engl J Med 2010 Nov 18; 363:1991.
PMID: 21083385
- Welsh MJ.
Targeting the basic defect in cystic fibrosis.
N Engl J Med 2010 Nov 18; 363:2056.
PMID: 21083391