The committee supports approving the CFTR modulator for patients 2 years and older with at least one non-class I CFTR gene mutation.

Cystic fibrosis is a life-threatening disease predominantly with respiratory symptoms. Most of the subjects with the disease harbor the F508del variant in the CFTR gene that causes the protein to ...

A real-world study in children with cystic fibrosis in the Netherlands found significant improvements in small airway function, BMI, and exacerbations after 12 months of treatment with ...