New and investigational treatments in cystic fibrosis

Cystic fibrosis (CF) is an autosomal recessive disorder that affects approximately 1 in 3000 Caucasian births, or 30,000 individuals in the US and 70,000 worldwide. The discovery of the CF gene, isolation of the CFTR protein and understanding of molecular mechanisms behind the clinical expression of CF are being translated into newer treatments. Treatments for CF and its manifestations are discussed in this article including inhaled antibiotics, hydrator therapies, anti-inflammatory agents and protein modifiers. New and experimental treatments that are in development are also discussed. Outcomes for these treatments are forced expiratory volume in one second (FEV 1 ) improvement, CF-related quality of life, use of intravenous antibiotics and frequency of exacerbations and hospitalizations.