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, but this can be disruptive for patients and their families, and does not reduce the risk of joint disease or death, the authors of the new study noted.
