Article
Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients.
Journal of internal medicine - 1 Feb 2015
Amaral M D
Abstract excerpt
Cystic fibrosis (CF) is the most common genetic life-shortening condition in Caucasians. Despite being a multi-organ disease, CF is classically diagnosed by symptoms of acute/chronic respiratory disease, with persistent pulmonary infections and mucus plugging of the airways and failure to thrive. These multiple symptoms originate from dysfunction of the CF transmembrane conductance regulator (CFTR) protein, a...
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