Cystic fibrosis is a life-limiting genetic disorder that affects several body systems including the lung (bronchiectasis), pancreas (malabsorption), liver (biliary cirrhosis), and sweat glands (heat shock). The cause is a mutation of the gene CFTR (cystic fibrosis transmembrane conductance regulator). Development and improved delivery of medications have resulted in a median life expectancy older than 40 years for this disorder once characterized as a pediatric illness. A recent Lancet article published online summarizes new therapies, that target CFTR, deemed effective in improving lung function and reducing exacerbations, as well as current research in small molecule and gene-based therapies.
Link to Lancet article abstract (04/29/2016)
