Results for search "Hemophilia".
Gene therapy shows promise in reducing, and even halting, potentially life-threatening bleeding events in people with hemophilia, researchers report.
Hemophilia A is the most common inherited bleeding disorder, affecting one in 5,000 males worldwide. It's caused by a missing coagulation factor called FVIII.
The current standard of care involves regular infusions of the FVIII protein...
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.
The early ...
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.
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