Cystic fibrosis transmembrane regulator potentiators as promising cystic fibrosis therapies

Expert Opinion on Investigational Drugs

Volumn 20, Issue 3, 2011, Pages 423-425

  Antoniu, Sabina Antonela ^a  

^a GRIGORE T POPA UNIVERSITY OF MEDICINE AND PHARMACY   (Romania)

Author keywords

class III mutation; cystic fibrosis; G551D; VX 770

Indexed keywords

CHLORIDE; ION TRANSPORT AFFECTING AGENT; PLACEBO; SODIUM; TRANSMEMBRANE CONDUCTANCE REGULATOR; UNCLASSIFIED DRUG; VX 770;

ADULT; CHLORIDE TRANSPORT; COUGHING; DIFFUSE MACULAR RASH; DOUBLE BLIND PROCEDURE; DRUG ERUPTION; DRUG SAFETY; DRUG WITHDRAWAL; FORCED EXPIRATORY VOLUME; GENE MUTATION; GLUCOSE BLOOD LEVEL; GLUCOSE URINE LEVEL; HUMAN; LUNG FIBROSIS; LUNG FUNCTION; MULTICENTER STUDY; NAUSEA; PAIN; PHASE 2 CLINICAL TRIAL; PROTEIN FUNCTION; REVIEW; RHINORRHEA; SIDE EFFECT; SODIUM TRANSPORT; TREATMENT RESPONSE;

ADOLESCENT; ADULT; AGED; AGED, 80 AND OVER; AMINOPHENOLS; CELL MEMBRANE; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DOUBLE-BLIND METHOD; HUMANS; ION TRANSPORT; MIDDLE AGED; MUTATION; QUINOLONES; YOUNG ADULT;

EID: 79951656025     PISSN: 13543784     EISSN: None     Source Type: Journal    
DOI: 10.1517/13543784.2011.554823     Document Type: Review

References (4)

1. Kalin N, Claass A, Sommer M, et al. DeltaF508 CFTR protein expression in tissues from patients with cystic fibrosis. J Clin Invest 1999;103(10):1379-89 (Pubitemid 29535494)
2. Accurso FJ, Rowe SM, Clancy JP, et al. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med 2010;363(21):1991-2003
3. Ratjen FA. Cystic fibrosis: pathogenesis and future treatment strategies. Respir Care 2009;54(5):595-605
4. Van Goor F, Hadida S, Grootenhuis PD, et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci USA 2009;106(44):18825-30