Article
Breakthrough therapies: Cystic fibrosis (CF) potentiators and correctors.
Pediatric pulmonology - 1 Oct 2015
Solomon George M, Marshall Susan G, Ramsey Bonnie W, Rowe Steven M
Abstract excerpt
Cystic Fibrosis is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene resulting in abnormal protein function. Recent advances of targeted molecular therapies and high throughput screening have resulted in multiple drug therapies that target many important mutations in the CFTR protein. In this review, we provide the latest results and current progress of CFTR modulators for...
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