Elexacaftor/VX-445–mediated CFTR interactome remodeling reveals differential correction driven by mutation-specific translational dynamics

Elexacaftor/VX-445–mediated CFTR interactome remodeling reveals differential correction driven by mutation-specific translational dynamics

Cystic fibrosis (CF) is one of the most prevalent lethal genetic diseases with over 2000 identified mutations in the cystic fibrosis transmembrane con…

European Commission approves expanded label for KAFTRIO

Vertex Pharmaceuticals has announced that the European Commission has granted regulatory approval for a label expansion of KAFTRIO in a combination regimen with ivacaftor for the treatment of people with cystic fibrosis.

#Advancing Cystic Fibrosis Treatment European Commission Approves KAFTRIO Label Expansion, Highlighting Vertex Pharmac...

:' The European Commission's recent regulatory approval for the expanded indication of KAFTRIO in combination with ivacaftor marks a significant development in the treatment paradigm for cystic fibrosis (CF). Vertex Pharmaceuticals Inc.'s continued innovation underscores its commitment to addressing unmet medical needs in CF populations, particularly for patients aged 2 years and older with at least one non-class I mutation in the cystic fibrosi…

New guidelines aim to boost equity in CF newborn screening in US

A set of new guidelines from the Cystic Fibrosis Foundation for CF newborn screening aims to improve equity in U.S. diagnosis and treatment.