Newer therapies for cystic fibrosis

Abstract 

Cystic fibrosis is the most common lethal inherited disease in Caucasians in the UK, with an incidence of approximately one in 2500 live births. It is a heterogeneous disease which reflects different mutations in the cystic fibrosis transmembrane conductance regulator gene, modifier genes and environmental influences. The median life expectancy of children with cystic fibrosis born in the UK is approximately 30 years. Longer survival is the result of improvements in basic therapies including airway clearance, aggressive use of antibiotics and optimizing nutrition. Care given in a specialist centre has also been shown to improve survival. Despite this progress, pulmonary disease still accounts for most of the morbidity and is the cause of death in over 90% of patients with cystic fibrosis. This review will focus on newer therapies aimed at pulmonary disease which are now being used in the clinical setting, and other novel therapies which are still at the research stage.

Keywords:  Cystic fibrosis, Cystic fibrosis transmembrane conductance regulator (CFTR), New therapies, Gene therapy