News

A new study sheds light on the challenges of diagnosing and managing gastrointestinal (GI) bleeding in individuals with severe von Willebrand disease…

CHOP Researchers Investigate Durability of Hemophilia A Gene Therapy Findings of a new preclinical study published online in Nature Communications may help address lingering…

On October 11, 2024, Pfizer announced that the U.S. FDA approved HYMPAVZI™ (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency…

 new preclinical study led by CHOP researchers, published in Nature Communications, offers promising insights into the long-term effectiveness of hemophilia A gene therapy….

The World Federation of Hemophilia (WFH) announced the 2025 World Hemophilia Day theme: Access for All: Women and Girls Bleed Too. Learn…

Phil Gattone said, “We greatly appreciate Pfizer’s innovative efforts in developing this novel treatment option that addresses some of the ongoing challenges…

Dive into NBDF’s August 2024 Government Relations Update for essential information on Medicaid unwinding, ACA premium tax credits, NIH reform, and state-level…

A manufacturing deviation at NovoNordisk’s Danish facility has led to potential underfilling of vials for NovoSeven©, NovoEight©, and Esperoct© – crucial treatments…

A session on von Willebrand Disease (VWD) at the National Bleeding Disorders Foundation Conference this past summer, highlighted patient-driven treatment advancements and…

A comprehensive document identifies 50 various publications about gene therapy, much of it accessible for parents and patients. The list includes: FAQs,…

Multimillion-dollar gene therapies were long awaited and desired, but patients were unsure whether insurance would cover the costs. So far, very few patients are…

These new medical-based webinars will speak to key topics relevant to the care of patients with inherited bleeding disorders. NBDF is excited…

Learn about Gene Therapy for Hemophilia with NBDF’s new resource collection. Find answers, research, and support. The National Bleeding Disorders Foundation (NBDF) is…

The National Bleeding Disorders Foundation (NBDF) is pleased to announce a new enduring and accredited educational activity available to healthcare providers. “Exploring…

BioMarin recently announced that they will be changing the scope of their efforts to manufacture and market their gene therapy product ROCTAVIAN.™…

by: Patricia Inácio, PhD | July 9, 2024 Hemgenix, the first approved hemophilia B gene therapy, was administered to two patients in France, marking the first time…

Gene therapy is a welcome treatment for patients, which has been anticipated for decades. But how do parents and patients discuss this…

NBDF’s National Youth Leadership Institute (NYLI) provides 18-24 year olds in the bleeding disorders community with leadership opportunities to encourage personal growth,…

While post-traumatic stress disorder is commonly connected with soldiers, people battling chronic illnesses can have it, too. Here’s how to identify the…

The National Bleeding Disorders Foundation (NBDF) announces the THRIVE campaign to raise awareness and educate on living healthily with bleeding disorders. The…

Pfizer recently provided new clinical trial updates for their investigational therapy marstacimab. The data was presented last month at the 2024 annual meeting…

In an NBDF interview, Bill Robie, Senior Director of Government Relations, highlighted the ongoing challenges of living with von Willebrand disease. In…

by: Patrizia Cavazzoni, MD, Director, Center for Drug Evaluation and Research and Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and…

Gene therapy research continues to evolve. As more information becomes available, the below may help you better understand gene therapy and shared…