title: From pipeline to patient: new developments in cystic fibrosis therapeutics.
abstract: BACKGROUND
  Cystic fibrosis (CF) is an inherited rare disease characterised by recurrent pulmonary infection, pancreatic malabsorption and a number of other multisystem effects. In the past few years new drugs specifically designed to treat CF have entered the pipeline, and some are now used in clinical practice.
  
  
  METHODS
  Clinical trial results from CF trials reported or ongoing within the last 3 years are discussed. A literature and trials registry search of trials and meta-analyses involving patients with CF was conducted, from which the most exciting developments were selected.
  
  
  CONCLUSIONS
  Drugs to address the basic defect in CF have finally come to market, albeit for a limited number of patients with a specific genotype. Traditional areas of CF therapeutics continue to be developed, with modest success, including drugs to modulate the airway surface liquid, new pancreatic supplements, antibiotics and new treatments for endocrine complications of CF.
authors: University of Nottingham, School of Clinical Science, Queens Medical Centre, Child Health, Nottingham, UK.
authors: Andrew P
authors: AP
authors: Prayle
authors: Alan R
authors: AR
authors: Smyth
mesh_terms: D000818:Animals; D000900:Anti-Bacterial Agents / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000503:physiopathology; D015195:Drug Design; D006801:Humans
chemical_list: D000900:Anti-Bacterial Agents