A gene-based therapy is potentially a step closer to becoming a one-time treatment for men with hemophilia. The life-threatening genetic disorder hinders the body’s ability to form blood clots.

Hemophilia and von Willebrand disease (VWD) are bleeding disorders that share similar symptoms. Both conditions make it more difficult for the blood to clot. Both conditions can also be genetic ...

Factor VIII gene was introduced into hematopoietic stem cells in men with hemophilia. The annualized bleeding rate went from 10 to 0. No worrisome gene integrations were seen. The content of this ...