I created Love To Breathe®️ in 2001 to be a positive voice in the CF community, to help educate and raise awareness about cystic fibrosis, and spread love whenever I can!
Wednesday, February 23, 2011
VERY good news....
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.