If you want to be in the know about what’s going on at our organization, you’ve come to the right place!
On December 4, 2025, Representatives Kean (R-NJ), Barragán (DCA), Miller-Meeks (R-IA), Auchincloss (D-MA), Fitzpatrick (R-PA), and Watson-Coleman (DNJ) reintroduced the Help Ensure Lower Patient (HELP) Copays Act to make necessary and life-saving prescription medications more affordable for patients with rare, serious, and chronic conditions such as hemophilia, von Willebrand disease, and other rare bleeding disorders.
CSL recently announced the publication of five years’ worth of data from their HOPE-B clinical trial program, which has been investigating the company’s hemophilia B gene therapy Hemgenix® (etranacogene dezaparvovec).
Federal:
Fiscal Year 2026 Funding Update
Star Therapeutics recently announced interim data from an ongoing phase 1/2 clinical trial evaluating its investigational von Willebrand disease (VWD) therapy VGA039.
For those of us who are able to give more, this is the perfect opportunity to get involved in your community and help spread the holiday cheer this season.
NBDF is excited to announce that its Facts About Inhibitors booklet is now available in an Arabic language version. Using easy-to-understand language, this publication includes important information about how inhibitors develop in people with hemophilia, key risk factors, and tips on getting screened.
The holiday season is a great time to recognize the many dedicated caregivers in the bleeding disorders community. This edition of the Resource Roundup (RR) includes resources that address everyday challenges universal to all caregivers as well as those unique to individuals with bleeding disorders.
If you want to join in and celebrate National Write a Friend Month during this wonderful winter season, here are a couple writing ideas to get you started.
With Thanksgiving nearly behind us and the winter holidays coming up, how can you make others feel warm during the cold season? We have some ideas to get you started.
NBDF’s inaugural Research Roundtable was held in Washington, D.C. on October 15-16, 2025. The meeting brought together lived experience experts (LEEs), researchers, healthcare providers, and industry leaders in a pre-competitive, product-agnostic setting to address barriers faced by women, girls, and people with the potential to menstruate in bleeding disorders research. Although the FDA could not attend due to the government shutdown, NBDF engaged with them on key agenda topics beforehand and plans to resume the dialog with them further once the government reopens.
The World Federation of Hemophilia (WFH) recently published an Arabic language version of their booklet Women and Girls with Hemophilia (2023) which is meant to aid outreach and educational efforts around the world, especially for such a classically underserved subgroup. Historically, women and girls affected by hemophilia have experienced protracted delays in securing an accurate diagnosis and care that takes bleeding disorders into full account.
Gratitude is about more than taking a second to say “Thanks.” Gratitude shows true appreciation. This Thanksgiving, we have some ideas for how you can give gratitude.
Pathway to Cures, NBDF’s venture philanthropy fund, has added SeraGene to their portfolio of emerging biotech companies addressing unmet needs in the inheritable blood and bleeding disorders community. Meet the team!
Three new documents have been issued by NBDF’s Medical and Scientific Advisory Council (MASAC), which creates recommendations and advisories on current treatment, research matters, and other general health concerns for the inheritable bleeding disorders community.
Off-Label Use of Emicizumab in Acquired Hemophilia A
Federal:
Government Shutdown
How can you spread the cheer by giving back? We have five altruistic ideas for the holiday season that just might do the trick.
The commercialization of hemophilia AAV gene therapies in recent years has created a steep learning curve for clinicians, patients, and other stakeholders in the bleeding disorders community. As a novel treatment unlike any that has come before, gene therapy has compelled a series of questions and considerations relevant to its efficacy, safety, patient eligibility, implementation, cost/reimbursement, and more.
This month looks at the world through the eyes of a child, so today we look at how you can recognize National Child Safety and Protection Month, and keep the children in your life safe.
NBDF monitors emerging therapies and changes in the therapeutic landscape that may affect people living with bleeding disorders. One of those developments involves BioMarin’s decision to out-license (allowing another company to market and sell) ROCTAVIAN, their gene therapy for adults with severe hemophilia A.
This statement from BioMarin explains their decision:
The latest Resource Roundup (RR) is dedicated to women, girls, and people with the propensity to menstruate (WGPPM), all historically underserved groups within the inherited bleeding disorders community. While recognition of these populations and their struggles to achieve healthy equity has increased recently, affected individuals continue to face challenges when it comes to timely diagnosis and management.
If you strive to live an altruistic lifestyle, there are lots of ways to help out this Halloween. Here are some top tricks to make Halloween a treat for everyone.
Tutors help students young and old to better their education, improve their skills and gain more success in a field of their choice. Why should you consider becoming a tutor?
Pathway to Cures, the venture philanthropy fund of the National Bleeding Disorders Foundation is focused on early-stage companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community. We carry out our work with volunteer expert advisors on our scientific advisory group, investment committee and board of directors. Today we spotlight our investment committee, our process for determining what is presented to this committee and the criteria used in making NBDF’s Pathway to Cures investments.
NBDF is pleased to inform U.S.-based clinicians and other interested individuals of a new series of virtual educational opportunities organized by our friends at the Irish Haemophilia Society (IHS). The IHS is hosting a series of clinical webinars on key topics developed for healthcare providers practicing in the global bleeding disorders community. The six webinars will be held on Zoom during the months of October, November, and December.
How can you encourage the children in your life to be active and volunteer with their community? We have some ideas for how to get kids involved in giving back.
There are plenty of ways to get involved in your community this month. To get you started, we came up with six spooky-fun volunteering ideas for October.
Star Therapeutics recently announced new financing (totaling $125 million) that will help the San Francisco-based biotechnology company continue to develop their investigational von Willebrand disease therapy VGA039. The U.S. Food and Drug Administration granted Fast Track designation to the experimental subcutaneous von Willebrand disease therapy (VWD) in January 2025.
Novo Nordisk has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration for Mim8 (denecimig), an investigational bispecific antibody therapy. The antibody promotes coagulation by binding two key proteins (factor IXa and factor X), essentially mimicking the function of the missing factor VIII in the clotting cascade.
If you’re a bookworm, you know just how great reading can be. If you’re not, check it out, and see all the benefits books can give.
While it is generally understood that the social determinants of health (SDOH) are significant factors contributing to an individual’s overall health, research specific to SDOH and its impact upon the bleeding disorders community has been rather limited. Authors of a newly published paper sought to address this knowledge gap by conducting a pilot study on SDOH in bleeding disorders, and gain insight on how these factors pose challenges among underserved groups in the U.S.
With the fall scholastic season now in full swing, we thought it would be a great time to share a few back-to-school resources to support students, families, and school personnel. This edition of the Resource Roundup features articles, educational videos and fact sheets, all of which speak to both school-based topics and bleeding disorders.
Make this season about more than just pumpkin spice lattes and costumes. Check out some of these ideas on how you can give back this month.
Five people who are inspired by the bleeding disorders community are lacing up for an immense physical challenge: running the TCS New York City Marathon on November 2, 2025. They’ll be running across the Verrazano-Narrows Bridge, through the streets of Brooklyn and Queens, and down 5th Avenue in Manhattan – and each step will help NBDF’s mission of helping people and families living with bleeding disorders to thrive.
Decades of advocacy spearheaded by the World Federation of Hemophilia (WFH) have resulted in a victory for the global bleeding disorders community. The World Health Organization (WHO) Model List of Essential Medicines (EML) has been updated to include safer and more effective medicines for the treatment of bleeding disorders.
Values and beliefs instilled now flourish into actions later. What can you do to instill philanthropic values in your children? To get you started, here are six ways to teach your child about giving.
The U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application for VONVENDI®, which expands its indication to include prophylaxis use to reduce the frequency of bleeding episodes in adults with von Willebrand disease (VWD), including those with type 1 and 2 disease, and for on-demand and perioperative management of bleeding in pediatric patients with VWD.
Federal:
2025 Bleeding Disorders Conference
NBDF wrapped up another successful Bleeding Disorders Conference. The policy team was busy with sessions on blood and product safety, access to insurance, and sharing updates on what’s happening in Washington, DC. This year there was also a session dedicated to NBDF’s advocacy efforts focused on women and girls. You can see the highlights from this year’s conference here.
Pathway to Cures, NBDF’s venture philanthropy fund, is an investor in Be Biopharma, Inc., a clinical-stage company developing engineered B cell medicines. Be Biopharma recently announced the first participant treated with their therapy BE-101 in the BeCoMe-9 Phase 1/2 clinical trial.
What is BE-101?
If you’re looking to celebrate this multicolor season in style with your family, check out these fun fall adventures you need to go on this year.
Sometimes we all need to feel like a kid again. If you’re tired of adulthood and want to rewind the clock, check out these six activities to relive those childhood memories.
Studies have found that volunteering can positively impact both your body and your mind. Here are a few ways doing good in your community can do some good for yourself!
Gene therapy was once hailed as the transformative breakthrough for hemophilia—the long-awaited cure that would change everything. But reality has been more nuanced, and many people in the community have been hesitant to embrace it. Some pharmaceutical companies have canceled their gene therapy programs, removing the treatment from the market. What this means for the future of innovation was what Friday's panel at NBDF's Bleeding Disorders Conference set out to explore.
Research, Evidence-based Practice, and QI: A Roadmap for Multidisciplinary Teams
Healthcare providers face a sobering reality: 400,000 unintended patient deaths occur annually, with adverse events affecting one in four hospital admissions. Yet these tragedies aren't due to incompetence—they can result from poorly-designed systems and inconsistent practices. Perhaps most striking, only 20-30% of healthcare decisions are based on solid evidence.
The last day of the 77th Annual Bleeding Disorders Conference brought inspiration at every turn—from sessions on data-driven research to stories from everyday heroes and celebrating leaders who make a difference. But the night was all about connection and fun.
The evening turned into a true celebration. With bohemian flair, live music, family games, great food, and plenty of dancing, the Final Night Event brought the energy and joy that made it the perfect ending to the conference.
Breakthrough sessions, hands-on workshops, and time together in community—Friday had it all. The day was full of learning, laughter, and moments that brought people closer.
One of the most meaningful parts of the Bleeding Disorders Conference was the chance for people to gather in spaces that reflected their identities and experiences. This year, NBDF hosted a series of intersectional sessions and networking hours that gave attendees the opportunity to sit together, share stories, and learn from one another. These gatherings honored the richness of our community while creating supportive spaces where everyone felt seen.
At NBDF's “Unlocking the Clotting Cascade,” at NBDF’s Bleeding Disorders Conference, attendees discovered that sometimes the best way to understand complex biology is to get your hands dirty—or in this case, get crafty with cotton balls and straws. In a hands-on workshop, participants rolled up their sleeves to build the clotting process piece by piece using everyday craft supplies.
We all set goals, and we don't always meet them. But what if there was a better way of thinking about goals? Rise and Thrive: Empowered Goal Setting, aimed to help attendees achieve their goals by thinking about them in a new way.
The workshop was hosted by Brendan Hayes, NBDF’s senior director of education, and summer intern Devin Boyle and was packed with practical tips and hands-on activities designed to help participants make real progress in their lives.
The treatment horizon for bleeding disorders looks remarkably different than it did just a few years ago. From nerve stimulation devices to injections every two months, from experimental pills to designer antibodies, the future of bleeding disorders care is unfolding with possibilities that extend far beyond traditional factor replacement. The popular, “Advancements in Treatments forn Bleeding Disorders,” took a look into the future.
Expanding Beyond Hemophilia
