This morning, the CFF announced very exciting results from the clinical study of a new treatment for cystic fibrosis. The oral drug, known as VX-770, targets the defective protein that causes CF.
A Phase 3 trial evaluated patients who carry at least one copy of a CF mutation called G551D.
Patients who took the drug showed marked improvements in a number of key measures of the disease, including improved lung function, fewer pulmonary exacerbations, patient-reported reductions in respiratory symptoms and weight gain.
In addition, average sweat chloride levels of patients on VX-770 dropped toward normal -- indicating the drug is impacting the underlying defect in CF.
VX-770 is being developed by Vertex Pharmaceuticals, and was discovered in collaboration with the CF Foundation.
I try to live each day to the fullest by keeping my hopes high, and dreams big!! I think it is important to LOVE as long as you LIVE, and LAUGH as much as you BREATHE!! I am a 37 year old living with Cystic Fibrosis, and an active advocate in the CF community. I make it a priority to raise awareness and help educate everyone on CF. Being diagnosed at 11 months old I was unable to make my own decisions. However, I am fortunate enough to have fabulous parents who chose to help fight my disease. When I was old enough I adopted my parents philosophy and joined the fight. This is my fight.....