a form of regenerative medicine. “It really is a medical milestone,” said Jeff Leiden, executive chairman at Vertex. “Suddenly, we have a different kind of therapy. You’re not just treatin ...

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated ... While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have ...

Two types of drugs improve CFTR function. Correctors bind to the protein and help it fold into the proper shape. Potentiators open channels of the protein. Vertex’s drugs combine both mechanisms ...

Pediatric cystic fibrosis patients treated using Vertex Pharmaceuticals’ oral CFTR potentiator VX-770 demonstrated significant improvements in lung function ... study will form part of the ...