期刊
EXPERT OPINION ON EMERGING DRUGS
卷 15, 期 4, 页码 653-659出版社
TAYLOR & FRANCIS LTD
DOI: 10.1517/14728214.2010.517746
关键词
CFTR pharmacotherapy; chloride secretion; clinical trials; cystic fibrosis; osmotic therapy
资金
- Inspire
Importance of the field: Cystic fibrosis (CF), one of the major respiratory diseases, is caused by mutations in a gene encoding for a chloride channel. Abnormal transepithelial ion transport leads to a reduced volume of the airway surface liquid layer and reduced mucociliary clearance. Areas covered in this review: There is currently no cure for CF and CF lung disease remains a major contributor to morbidity and mortality. However, most current treatments for CF lung disease do not address the underlying pathology. We describe here new therapeutic developments aiming to identify or generate compounds that counteract the effects of cystic fibrosis transmembrane conductance regulator (CFTR) on the airway. What the reader will gain: This review summarizes the current state of new developments in the treatment of CF lung disease. These drugs include nebulized and inhaled osmotically active agents, but also modifiers of ion channels other than CFTR, such as activators of alternative chloride channels or inhibitors of sodium absorption, and compounds in development aim to correct or improve impaired CFTR function directly. First clinical trials with new drugs including ion channel modifiers and CFTR pharmacotherapeutics have revealed very promising results. Take home message: CF drug therapy is moving rapidly from symptomatic therapy to treatment of the underlying pathophysiology.
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