Cystic fibrosis

Cystic fibrosis (CF) is the most common lethal genetic disease in the Caucasian population. Survival has improved dramatically with expected median survival in the UK now 41.4 years. In CF, a vicious cycle of mucus stasis, chronic inflammation and recurrent infection leads to respiratory failure and death in the majority of patients. CF is a multisystem disease with pancreatic exocrine insufficiency occurring in the majority of patients.Treatment involves airway clearance techniques, inhaled muco-active therapies and the prevention and control of respiratory infection. Pseudomonas aeruginosa is the most common respiratory pathogen.In 2011, a mutation-specific therapy (VX-770) demonstrated efficacy for the first time. A number of other similar compounds are in the pipeline. With these treatments we are potentially entering a new era of CF care in which we may expect even longer survival.