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.
To read more about VX-770 click here
2 comments:
yayyyy! That is fabulous news!! That brings a smile to my face! I am sure this is only the beginning too...
I read about that too - very good news!!
Post a Comment