Cystic Fibrosis: New therapies developed

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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)

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