Sharma J, et al: A novel G542X CFTR rat model of cystic fibrosis is sensitive to nonsense mediated decay ... and additional approaches for rare CFTR mutations not yet addressed Core Function #2: ...

A new mega-database of half a million mutations may flag new ways of treating genetic disease, scientists say.

inhibition because the G542X mutation was introduced into the mouse Cftr locus and thus the transcript is spliced normally and is subject to NMD. CF null (CFTR-/-) rat: The first CF rat model, ...

In 1997, the National Institutes of Health convened a Consensus Development Conference on Cystic Fibrosis (CF). 1 The Consensus Conference recommended that genetic screening for CF mutations ...

An international study has identified two types of these so-called dysbioses in cystic fibrosis. They display ... Landmark Study Involving Babies in Ireland Supports Use of Cystic Fibrosis Drug in ...

According to Vertex Pharmaceuticals, approximately 300 additional people with CF are now eligible to receive treatment.

for cystic fibrosis (CF) in adults and children 6 years and up, drugmaker Vertex Pharmaceuticals announced on Friday. Eligible patients must have at least one mutation in F508del or another ...

Initially, CF was thought to be caused by nutritional deficiencies, persistent infection, and many others —until in 1989, ...

The FDA has approved Vertex’s Alyftrek, a once-daily triple combination for the treatment of cystic fibrosis in people 6 years and older who have mutations that are amenable to the therapy ...

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' next-generation treatment for a rare and progressive genetic disease, the company said on Friday, expanding its dominance in ...