title: New cystic fibrosis drug paves the way for orphan diseases.
authors.forename: David
authors.initials: D
authors.lastname: Cooney
mesh_terms: D000627:Aminophenols / Q000191:economics / Q000494:pharmacology; D003550:Cystic Fibrosis / Q000188:drug therapy / Q000191:economics / Q000235:genetics; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D017277:Drug Approval; D016527:Drug Costs; D006801:Humans; D009154:Mutation; D057285:Precision Medicine / Q000191:economics / Q000379:methods; D015363:Quinolones / Q000191:economics / Q000494:pharmacology; D035583:Rare Diseases / Q000191:economics*; D019141:Respiratory System Agents / Q000191:economics / Q000494:pharmacology; D006113:United Kingdom
chemical_list: D000627:Aminophenols; C505032:CFTR protein, human; D015363:Quinolones; D019141:Respiratory System Agents; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor