“A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate.
Patients who received the one-time intravenous therapy continue to have a more than 90 percent decrease in bleeding events two to three years after their initial treatment, researchers reported Jan. 1 in the New England Journal of Medicine.
The therapy fixes a broken gene in liver cells that causes production of flawed factor VIII, a protein that plays a key role in blood clotting.
People with this genetic mutation have hemophilia A, the most common type of this bleeding disorder. Hemophilia A accounts for 8 out of 10 cases of hemophilia, researchers said.”