helping reduce the symptoms of cystic fibrosis. However, these medications are only effective in people with specific CFTR mutations. Therefore, doctors will choose the type of CTFR modulator ...

Eric Sorscher and colleagues at UAB developed two different ways to overcome the genetic mutations responsible for cystic fibrosis. Cystic fibrosis is a hereditary disease caused by mutations in a ...

CF often arises from mutations that make the cystic fibrosis transmembrane conductance regulator (CFTR) protein misfolding and malfunction, causing thick mucus to aggregate in the lungs while also ...

Single gene disorders, like Huntington’s disease and cystic fibrosis ... have shown that mutations in additional genes could perhaps modulate the severity and type of disease-related phenotypes ...

About Cystic Fibrosis Cystic fibrosis (CF) is a progressive genetic disease that causes persistent lung infections and respiratory failure. CF is caused by mutations in the cystic fibrosis ...

The U.S. Food and Drug Administration has approved Vertex Pharmaceuticals’ ALYFTREK™ for treating cystic fibrosis in individuals aged six and older with specific gene mutations. This approval ...