title: Cystic fibrosis transmembrane regulator potentiators as promising cystic fibrosis therapies.
abstract: BACKGROUND
  Several types of mutations in the cystic fibrosis transmembrane regulator (CFTR) gene lead to abnormal CFTR protein and alterations of chloride and sodium transmembrane transportation in cystic fibrosis (CF). Some investigational compounds such as VX-770 can improve CFTR protein function.
  
  
  METHODS
  This paper discusses the results of a Phase II study investigating the safety and efficacy of VX-770 in patients with CF.
  
  
  CONCLUSIONS
  VX-770 is able to improve chloride and sodium transportation and has a good safety profile. Although such compounds have limited therapeutic targeting potential, preliminary results show great promise in the context of CF therapy.
authors.affiliation: University of Medicine and Pharmacy Gr. T. Popa Iasi, Pulmonary Disease University Hospital, Department of Internal Medicine II-Pulmonary Disease, 30 Dr I Cihac Str., Iasi, Romania. sabina.antonela.antoniu@pneum.umfiasi.ro
authors.forename: Sabina Antonela
authors.initials: SA
authors.lastname: Antoniu
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D006801:Humans; D008875:Middle Aged; D055815:Young Adult; D000627:Aminophenols / Q000009:adverse effects / Q000627:therapeutic use; D002462:Cell Membrane / Q000378:metabolism; D003550:Cystic Fibrosis / Q000188:drug therapy / Q000235:genetics / Q000378:metabolism; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics / Q000378:metabolism; D004311:Double-Blind Method; D017136:Ion Transport / Q000187:drug effects; D009154:Mutation; D015363:Quinolones / Q000009:adverse effects / Q000627:therapeutic use; D017322:Clinical Trials, Phase II as Topic; D015337:Multicenter Studies as Topic; D018849:Controlled Clinical Trials as Topic
chemical_list: D000627:Aminophenols; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor; D015363:Quinolones