In terms of hemophilia gene therapy, the authors assert the need for more favorable risk/benefit ratios especially considering the “one-and-done” nature of currently FDA approved AAV-vectors based therapies.

BE-101 is engineered to insert the human factor IX (FIX) gene into primary human B cells and initiate the continuous production of therapeutic levels of the FIX protein.

The esteemed researcher joins the Scientific Advisory Group

New name reflects community leadership and a shift from planning to action

Immune thrombocytopenia (ITP) is a rare autoimmune disorder that causes a significant reduction in platelets, which are necessary for clotting.

A comprehensive literature review was conducted to ensure that the von Willebrand disease guidelines were still clinically appropriate and informed by the research described in more current peer-reviewed papers.

HYMPAVZI™ (marstacimab-hncq) is FDA-approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric individuals 12 years of age and older with hemophilia A without factor VIII inhibitors, or hemophilia B without factor IX inhibitors

NFRC is highlighting resources to encourage better joint health in people with hemophilia and other bleeding disorders.

This activity explores illustrative case studies, presentations, and thoughtful discussions about the critical need for regular screening for anxiety/depression and bone health in women with inherited bleeding disorders.

Dr. Kasper was a pioneer in bleeding disorders treatment and research and a lifelong champion for the community.