Hemophilia

Phase III trial shows 86% reduction in treated bleeds in hemophilia A and B with inhibitors

First gene therapy approved for severe hemophilia A

News, features, and commentary about cancer-related issues

Factor VIII replacement therapy provides protection from bleeds with once-weekly dosing

Model predicts valoctocogene roxaparvovec could maintain mild hemophilia levels for 5 years

Weekly dosing with efanesoctocog alfa reduced annualized bleeding rate by 77%

Hemgenix reduced rate of annual bleeds, eliminated need for factor IX prophylaxis therapy

Gene therapy continues to show promise in clinical trials, but is it ready for the clinic?

"This is the most exciting time ever" to be a hematologist, says ASH President Jane Winter, MD

Eric Winer, MD, talks about how growing up with hemophilia helped him connect with patients

Small study suggests normal levels can be achieved with low vector doses of FLT180a

Valoctocogene roxaparvovec significantly increased factor VIII activity, reduced bleeding

Monthly infusion vs on-demand bypassing agents effective in patients with or without inhibitors

Most patients do well in 3-year phase I/II study