New therapies in cystic fibrosis.

Abstract:

: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.

journal_name

Curr Pharm Des

authors

Ratjen F,Grasemann H

doi

10.2174/138161212799315984

subject

Has Abstract

pub_date

2012-01-01 00:00:00

pages

614-27

issue

5

eissn

1381-6128

issn

1873-4286

pii

BSP/CPD/E-PUB000881

journal_volume

18

pub_type

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