Please provide your email address to receive an email when new articles are posted on . Patients given aerosolized 4D-710 had elevated CFTR protein levels that went over normal levels. Over 12 months, ...

Previously disclosed post hoc PreciSION CF analyses of SION-719 support advancement of SION-451 + SION-2222 into the AscenSION CF Phase 2a proof-of-concept trial ...

Cystic fibrosis (CF) affects over 100,000 people worldwide and is caused by one of several known mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. The CFTR protein is a ...

The process of generating proteins from genes is akin to a factory, where workers follow a set of instructions that, ideally, are effective and clear. But for some people who suffer from cystic ...

A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers have succeeded in developing a so-called nanobody that penetrates directly ...

New combinations of molecules put many patients on the path to good health. But those with rarer mutations are, for now, left behind. Over the course of two decades spent developing treatments for the ...

Despite remarkable medical advances over the last years, cystic fibrosis remains the most prevalent lethal genetic disease. It is due to mutations in the CFTR protein which is normally required to ...

An experimental drug suggests that a 'path is clearly achievable' to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent of cystic ...

Protein misfolding is a likely culprit in many degenerative disorders. Cystic fibrosis, for instance, is caused by mutations in the CFTR gene that prevent the eponymous protein from assuming its ...

Researchers have identified hundreds of new proteins that could play a role in cystic fibrosis, and which may shed light on why some patients respond better than others to current therapies.

Medically reviewed by Reza Samad, MD Key Takeaways Cystic fibrosis treatment requires airway clearance techniques to remove ...