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Current Pharmaceutical Design


ISSN (Print): 1381-6128
ISSN (Online): 1873-4286

New Therapies in Cystic Fibrosis

Author(s): Felix Ratjen and Hartmut Grasemann

Volume 18, Issue 5, 2012

Page: [614 - 627] Pages: 14

DOI: 10.2174/138161212799315984

Price: $65


Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that lead to abnormalities in transepithelial ion transport in the airways of affected patients. Lung disease is the major contributor to morbidity and mortality in patients with cystic fibrosis but recommended therapeutic interventions so far have focussed on symptom control rather than treatment of the underlying causes of the disease. New therapies that are currently in pre-clinical and clinical testing include CFTR pharmacotherapy, drugs targeting other ion channels, and hydrators of the cystic fibrosis airways. The current status of these and other new developments in the treatment of cystic fibrosis are reviewed

Keywords: Cystic fibrosis, lung disease, therapy, mutations, transmembrane conductance regulator (CFTR) gene, airways, mucus retention, bacterial infection, neutrophilic inflammation, osmotic gradient

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