An experimental injectable drug appears effective in reducing bleeds in patients with hemophilia A and B, according to a pair of new clinical trials.
Two-thirds of people with treatment-resistant hemophilia who were treated with the drug fitusiran had no bleeds at all after nine months, versus just 5% of people treated with drugs that enhance clotting, according to a trial published onlin...
Researchers may have found a way for people with severe hemophilia to take their standard treatment less often, if the results of an early trial pan out.
In what experts called a feat of bioengineering, scientists were able to create a "fusion protein" that may extend the interval between treatments for hemophilia -- from about every couple of days to once a week.
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% decrease in bleeding events two to three years after their initial treatment, researchers reported Jan. 1 in the New England Journal of Medicine.