News
Investigational Sub-Q von Willebrand Disease Therapy Put on Fast Track
Industry News & Research
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the investigational von Willebrand disease (VWD) therapy VGA039. The…
Clinical Practice and Hemophilia Gene Therapy the Focus of New Accredited Activity
Gene Therapy, Industry News & Research
The National Bleeding Disorders Foundation (NBDF) is excited to announce a new online, enduring, and accredited educational activity available to healthcare providers….
Washington Days 2025, NBDF’s Annual Advocacy Event, is March 5-7
Advocacy & Legislation, Industry News & Research
Are you ready to advocate for the bleeding disorders community? Join us at Washington Days 2025 for a powerful and impactful experience. Learn…
Learn About the T.H.R.I.V.E. Campaign
Health and Well Being, Industry News & Research
The T.H.R.I.V.E. campaign is an initiative to educate individuals with bleeding disorders and their support networks about six key steps for leading…
How to Protect Yourself from Fractures
Health and Well Being, Industry News & Research
Bone health isn’t necessarily top of mind — especially at younger ages — but it should be. One study revealed that people with…
Beyond Medication: The Benefits of Physical Therapy for Chronic Pain
Industry News & Research, Pain Management
Chronic pain affects up to 58% of people with hemophilia, impacting mobility, mental health and daily activities. Recognizing this widespread issue, the…
New Recommendations Issued on Bleeding Disorders Treatment, Bone Health and Mental Health
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
The National Bleeding Disorders Foundation (NBDF) has announced that three new documents have been issued by the Medical and Scientific Advisory Council…
B Informed!
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
For thirty years, the Coalition for Hemophilia B has improved the quality of life for people with hemophilia B and their families…
New Study Looks at GI Bleeding in von Willebrand Disease
Industry News & Research
A new study sheds light on the challenges of diagnosing and managing gastrointestinal (GI) bleeding in individuals with severe von Willebrand disease…
Durability of FactorVIII Focus of New Hemophilia A Gene Therapy Study
Industry News & Research
CHOP Researchers Investigate Durability of Hemophilia A Gene Therapy Findings of a new preclinical study published online in Nature Communications may help address lingering…
FDA Approves Pfizer’s HYMPAVZI for Hemophilia A and B Treatment
Industry News & Research
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 Study Uncovers Breakthrough for Long-Term Hemophilia A Gene Therapy
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
new preclinical study led by CHOP researchers, published in Nature Communications, offers promising insights into the long-term effectiveness of hemophilia A gene therapy….
2025 World Hemophilia Day Theme Revealed
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
The World Federation of Hemophilia (WFH) announced the 2025 World Hemophilia Day theme: Access for All: Women and Girls Bleed Too. Learn…
FDA Approves Pfizer’s HYMPAVZI for Hemophilia A and B Treatment without Inhibitors
Industry News & Research
Phil Gattone said, “We greatly appreciate Pfizer’s innovative efforts in developing this novel treatment option that addresses some of the ongoing challenges…
Critical Policy Updates: Medicaid Changes, ACA Credits, and More
Advocacy & Legislation, Health and Well Being, Industry News & Research
Dive into NBDF’s August 2024 Government Relations Update for essential information on Medicaid unwinding, ACA premium tax credits, NIH reform, and state-level…
Product Update from NovoNordisk
Industry News & Research
A manufacturing deviation at NovoNordisk’s Danish facility has led to potential underfilling of vials for NovoSeven©, NovoEight©, and Esperoct© – crucial treatments…
VWD: A Glimpse into Tomorrow’s Treatments
Industry News & Research, Living with a Bleeding Disorder
A session on von Willebrand Disease (VWD) at the National Bleeding Disorders Foundation Conference this past summer, highlighted patient-driven treatment advancements and…
Learn More About Gene Therapy
Gene Therapy, Health and Well Being, Industry News & Research
A comprehensive document identifies 50 various publications about gene therapy, much of it accessible for parents and patients. The list includes: FAQs,…
Gene Therapy’s Slow Start
Gene Therapy, Health and Well Being, Industry News & Research
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…
Attention Clinicians! New Bleeding Disorders Education Available from IHS
Industry News & Research
These new medical-based webinars will speak to key topics relevant to the care of patients with inherited bleeding disorders. NBDF is excited…
New Resource Compilation on Gene Therapy for Hemophilia
Gene Therapy, Industry News & Research
Learn about Gene Therapy for Hemophilia with NBDF’s new resource collection. Find answers, research, and support. The National Bleeding Disorders Foundation (NBDF) is…
NBDF Announces a New Enduring and Accredited Educational Activity Available to Healthcare Providers
Industry News & Research
The National Bleeding Disorders Foundation (NBDF) is pleased to announce a new enduring and accredited educational activity available to healthcare providers. “Exploring…
BioMarin to Narrow Its Commercial Focus for Hemophilia A Gene Therapy
Gene Therapy, Industry News & Research
BioMarin recently announced that they will be changing the scope of their efforts to manufacture and market their gene therapy product ROCTAVIAN.™…
France Gets Gene Therapy
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
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…