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Treatment strategies for cystic fibrosis: what's in the pipeline?

Cystic fibrosis (CF) is an inherited disease, characterised by pancreatic malabsorption (with poor growth) and impaired mucociliary clearance (leading to recurrent pulmonary infection). The prognosis has markedly improved over the last 40 years. This was achieved initially by improving pancreatic enzyme supplements and more recently through drugs designed to treat pulmonary infection, reduce inflammation and enhance mucociliary clearance. The CF gene has been sequenced, allowing new therapeutic targets to be identified. Currently the greatest therapeutic challenges are to correct the biochemical defect of CF and to identify effective treatments for chronic respiratory infection. This review provides an overview of CF, the treatment strategies currently used, and new drugs undergoing clinical trials.