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The U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (Pfizer, Inc.), a new hemophilia B gene therapy product. It is designed with bioengineered adeno-associated virus (AAVs) vectors to introduce a working copy of the factor IX gene. The therapy is administered as a one-time intravenous infusion to stimulate the long-term production of clotting factor IX (FIX) and prevent bleeding. This represents the second hemophilia B gene therapy product to be approved in the U.S ..read more

New documents issued by NBDF’s Medical and Scientific Advisory Council (MASAC) cover a wide range of topics relevant to bleeding disorder patients in the U.S. and abroad. They encompass recommendations for treatment with licensed therapies, chronic pain management and physical therapy, gene therapy terminology, and bleeding disorders diagnosis in women and girls. Also included are two new resolutions challenging international recommendations that could contribute to negative health and quality of life implications for patients.  ..read more

On April 2, 2024, 8 News Now covered our Las Vegas Chapter's Winter Wine Walk with Stephanie Dupree ..read more

Data provided by the National Bleeding Disorders Foundation (NBDF) was referenced recently in the coverage of a tragic case in Chelsea that took a dark turn for 3-year-old Yael Guardado Prudencio, a child with hemophilia and a seizure disorder. This incident has raised questions about the challenges faced by families on society's margins and the consequences of leaving vulnerable children without proper care ..read more

For Immediate Release  CONTACT:   ..read more

Financing a higher education poses considerable challenges, especially as yearly tuition and other school-related costs continue to rise. For many individuals and families considering a higher education, awareness of any opportunities to defray those costs is critical. In addition, because decisions about school can have both near-term and long-term implications, it is also helpful to understand the many financial variables.    ..read more

U.S. Hemophilia Treatment Centers (HTCs) are once again participating in the National HTC Patient Satisfaction Survey (PSS). It is launched every three years to help glean valuable feedback from HTC patients and caregivers, with the overarching goal of enhancing care. Participants in the PSS often include people with hemophilia, von Willebrand disease, and other inherited bleeding disorders.   ..read more

A new paper published in The Lancet Haematology journal includes data from the HOPE-B clinical trial program that is investigating the hemophilia B gene therapy etranacogene dezaparvovec – the product’s commercial name is Hemgenix® (CSL Behring).   ..read more

Keeping Your Informed  ..read more