title: Cystic Fibrosis: A Novel Pharmacologic Approach to Cystic Fibrosis Transmembrane Regulator Modulation Therapy.
abstract: Therapy for cystic fibrosis (CF) has progressed during the past several decades. Much of this progress is because of advances in genetic testing to precisely identify the underlying cause of CF transmembrane regulator (CFTR) dysfunction. However, with more than 1900 mutations that can produce a faulty CFTR, the management of CF can remain a challenge. Several innovative drugs recently approved by the Food and Drug Administration, termed genetic modulators, target the underlying disease by modulating the CFTR defect. This review provides physicians with an established simple classification scheme to guide their use of these drugs. The treatment challenge of 1900 CFTR mutations has been simplified into 6 physiologic classes, each paired with an available therapy to offer patients the most functional improvement. Drug therapy monitoring, adverse effects, and indications for discontinuation must also be considered.
authors: Deborah
authors: D
authors: Virant-Young
authors: Justin
authors: J
authors: Thomas
authors: Sarah
authors: S
authors: Woiderski
authors: Michelle
authors: M
authors: Powers
authors: Joseph
authors: J
authors: Carlier
authors: James
authors: J
authors: McCarty
authors: Tyler
authors: T
authors: Kupchick
authors: Anthony
authors: A
authors: Larder
mesh_terms: D000627:Aminophenols / Q000627:therapeutic use*; D000631:Aminopyridines / Q000627:therapeutic use*; D052117:Benzodioxoles / Q000627:therapeutic use*; D065101:Chloride Channel Agonists; D003550:Cystic Fibrosis / Q000188:drug therapy / Q000235:genetics / Q000378:metabolism; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000187:drug effects / Q000235:genetics / Q000378:metabolism; D004247:DNA / Q000187:drug effects / Q000235:genetics*; D004252:DNA Mutational Analysis; D006801:Humans; D009154:Mutation / Q000187:drug effects*; D010069:Oxadiazoles / Q000627:therapeutic use*; D015363:Quinolones / Q000627:therapeutic use*
chemical_list: D000627:Aminophenols; D000631:Aminopyridines; D052117:Benzodioxoles; C505032:CFTR protein, human; D065101:Chloride Channel Agonists; D010069:Oxadiazoles; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor; D004247:DNA; C569105:lumacaftor; C515878:ataluren