This past week saw the approval of two new therapies, representing significant scientific breakthroughs in the field of cell-based gene therapy to treat patients with sickle cell disease (SCD), a rare condition historically associated with few…
CSL Behring recently announced three-year post treatment data from clinical trial participants who received a one-time infusion of Hemgenix® (etranacogene dezaparvovec), an adeno-associated virus (AAV) vector-based hemophilia gene therapy. Hemgenix…
The National Blood Disorders Foundation (NBDF) and the American Thrombosis & Hemostasis Network (ATHN) are collaborative partners that share many key aspects of their missions, most importantly, improving the lives of people affected by…
Pfizer Inc. recently announced that the U.S. Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) for marstacimab, its investigational hemophilia therapy that targets an anticoagulant protein known as tissue factor…
Researchers at the Yale School of Medicine have been studying the key clotting protein von Willebrand factor (VWF), to shed light on the processes by which it is stored and released in response to an injury. As VWF is the deficient protein in many…
Though it is generally recognized in the bleeding disorders community that physical therapists (PTs) are essential members of the core interdisciplinary teams of U.S. hemophilia treatment centers (HTCs), understanding of their educational/resource…
Washington Days: Registration for Washington Days is now open and closes on January 15th. Register today to join advocates nationwide in Washington D.C. on March 6-8, 2024 to advocate for the HELP Copays Act as well as key…
Join us to hear the results of a Pilot Study of US HTC Physical Therapists' Concordance of PT MASAC Recommendations and Educational Needs.
NBDF's Wednesday Webinars are a free education series open to providers and community members.…
Formulary restrictions are one of the primary access barriers to inheritable blood and bleeding disorders treatment. With health plan coverage changes happening again this year, join NBDF and HFA to learn about these restrictions, how we'…