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Gene Therapy Could Be Big Advance Against 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...

Early Trial Offers New Hope for People With Hemophilia

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 ...

Gene Therapy May Be Long-Term Cure for Type of Hemophilia

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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