title: [FUTURE THERAPIES FOR CYSTIC FIBROSIS].
abstract: Current cystic fibrosis treatment is symptomatic and has greatly increased life expectancy over the last decades. However, mean age of death is still 29-years-old. Innovative therapeutic molecules aiming at restoring CFTR function are developed. Ivacaftor is the first medicine approved in 2012 for the 3% of patients worldwide carrying the rare CFTR mutation called G5510. Ivacafter is associated with a sustained improvement in respiratory function and a decreased frequency of pulmonary exacerbations. Other pharmacotherapies are being developed that aim at correcting other CFTR defects due to other CFTR mutations, especially the most frequent F508del mutation.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Fajac
mesh_terms: D000627:Aminophenols / Q000627:therapeutic use*; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D006801:Humans; D015363:Quinolones / Q000627:therapeutic use*; D035703:Therapies, Investigational
chemical_list: D000627:Aminophenols; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor