Cysts, fibrosis, and tubular degeneration form the typical triad of pathologies leading to the gradual decline in kidney ...

While many of the patients that come through the UCSF Fresno Cystic Fibrosis Center are still young, they have transitioned ...

Investigators from Mass General Brigham and Beth Israel Deaconess Medical Center have developed STITCHR, a new gene editing ...

Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...

The new approval makes the regimen an option for around 4,000 patients in the EU who have at least one non-class I CFTR mutation.

Vertex continues to capitalize on its leadership in cystic fibrosis (CF) treatments, where its blockbuster drug Trikafta is ...

Good morning! Citigroup in the middle of funding freeze fallout, Ursula von der Leyen offers Trump a “zero-for-zero” tariffs ...

Vertex Pharmaceuticals (VRTX) announced that the European Commission has granted regulatory approval for a label expansion of KAFTRIO in a ...

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

A revolution is underway in gene editing -- and at its forefront is David Liu, an American molecular biologist whose ...

New U.S. guidelines call for major updates to cystic fibrosis newborn screening protocols, aiming to improve accuracy, ...

"Newborn screening for cystic fibrosis has been universal in the U.S. since 2010; however, there is significant variation ...