Vertex Pharmaceuticals (VRTX) stock in focus as company secures EU backing to expand label for its cystic fibrosis therapy ...

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

Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the ...

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 ...

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat ...

Sionna Therapeutics found strong investor interest in its cystic fibrosis drugs, which could rival blockbuster Vertex ...

NBD1 has long been thought to be important for normalizing CFTR function because it’s the location of the F508del mutation — the most common mutation that causes CF. Sionna aims to succeed ...

The management of CF has been changing drastically over the last few years, especially after the introduction of CFTR modulators. Currently, 188,336 people have the disease in 96 countries, but only ...