Cystic fibrosis (CF) patients treated with Kalydeco (ivacaftor) who have the G551D mutation reported better health-related quality of life (HRQoL) and symptom relief than those on standard treatment who carry the F508del mutation in the CFTR gene, according to a real-world study. The research, “Patient-reported…

A new report on cystic fibrosis (CF) in England and Wales further supports evidence of the groundbreaking impact that Kalydeco (ivacaftor) has had on treating CF, and reinforces the Cystic Fibrosis Trust‘s urgency for the drug to be made available to the 44 patients with rare CF gene mutations in…

Vertex Pharmaceuticals Incorporated has been grabbing headlines recently related to its revolutionary cystic fibrosis drug Kalydeco (ivacaftor), which is currently approved for people with CF aged 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Clinical-stage biotech company Galapagos, which is based in Belgium, has announced positive results of a Phase II clinical trial for two oral drugs designed to treat the main mutations in cystic fibrosis (CF) patients, including F508del and G551D. AbbVie has been working in collaboration with Galapagos in order to further the development…

In some ways, it’s both the best of times and the worst of times for persons afflicted with certain serious disease conditions, such as cystic fibrosis. On the one hand, there have been encouraging and potentially life-changing breakthroughs in terms of therapy options. On the other, these new drugs can…

Adding to the excitement of Vertex Pharmaceuticals Inc.‘s new orphan drug status of VX-661 is the announcement of the results of Vertex’s Phase 2 clinical study of…