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

Check out the highlights in photos from day one!

Defining Hope, Strength, and Infinite Possibility

Community member Ryan Warnall's voice filled the auditorium as he performed "The Impossible Dream," setting an emotional tone that would define the National Bleeding Disorders Foundation (NBDF) Opening Session on Thursday, August 21, 2025. His performance captured something essential about this community: a group that has repeatedly turned the impossible into reality, from fighting for a safer blood supply to developing treatments that seemed beyond hope decades ago.

NBDF’s Bleeding Disorders Conference welcomed many people new to bleeding disorders, and offered preconference sessions to help these new attendees acquire some basic knowledge to assist them on their education journey throughout the conference. “Hemophilia Treatment Landscape,” on Thursday, August 21, 2025, gave attendees a comprehensive overview in the current ways of treating hemophilia. 

Why does treatment for women and girls with bleeding disorders lag so far behind treatment for men, and what can we do about it? These were the central questions of “Women Bleed Too: Addressing the Blind Spots,” on Thursday, August 21, at NBDF’s Bleeding Disorders Conference.

On Thursday evening, from 6:30 to 7:30 pm in the Exhibit Hall, NBDF held a special “Meet the Poster Abstract Authors” session, featuring the 47 poster abstract approved for display during this year’s BDC in Aurora, Colorado. All conference attendees had an opportunity to view the posters and meet with the authors directly.  The authors represent universities, pharmaceutical companies, hemophilia treatment centers, or other bleeding disorders organizations in the US or beyond.   Abstracts were initially submitted to NBDF in spring and then assessed by a panel of experts.

The National Bleeding Disorders Foundation’s (NBDF) 77th Annual Bleeding Disorders Conference (BDC) is finally here. We like to think of this as a family reunion – whether you’re new to the community or have been attending for years, you’ll be welcomed with open arms.

Welcome to the Heart of Our Community

For people who have just discovered that they have VWD, the diagnosis can seem overwhelming. The Thursday pre-conference session, “VWD: Your Masterclass in VWD Insights and Community,” sponsored by CSL Behring, was designed to give attendees a firm understanding of the bleeding disorder, current treatments, and what future therapies are being studied.

Big news! NBDF’s Pathway to Cures portfolio company Spark Biomedical was selected by Wellcome Leap as an awardee of “The Missed Vital Sign” worldwide program.

Concizumab-mtci (Alhemo®) has received approval from the US Food and Drug Administration (FDA) as a once-daily prophylactic treatment for patients aged 12 years and older with hemophilia A or B without inhibitors.

This new approval results in an expanded indication for Concizumab-mtci, which was initially approved by the FDA in December 2024 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.

Explore the latest updates on federal budget reconciliation, healthcare lawsuits, and state-level policy changes impacting bleeding disorders. Learn how NBDF is advocating for access, funding, and patient protections.

Federal: 

At the turn of the 21st century, there was great excitement and anticipation of gene therapy as a ‘curative’ treatment for hemophilia A and B patients, particularly among those with moderate-to-severe forms of the disease. However, a recent forum piece in the journal Research & Practice in Thrombosis & Haemostasis (RPTH) offers some reasons why the overall commercial uptake of these therapies has been relatively modest, as lingering questions over long-term efficacy (in the case of hemophilia A) and longer-term safety of these first-generation gene therapies.

Background 

According to multiple news outlets, the U.S. Centers for Medicare & Medicaid Services (CMS) in mid-July provided the personal data of millions of Medicaid enrollees to the U.S. Department of Homeland Security (DHS), as part of the Administration’s ongoing immigration enforcement efforts. This action marked a change from longstanding policy and previous practice barring such information sharing. 

Clinical stage biotechnology Be Biopharma, Inc. recently announced that the first hemophilia B patient has been treated with a single intraveneous dose of their investigational cell therapy, BE-101. The ongoing BeCoMe-9 Phase 1/2 study is evaluating the safety and efficacy of BE-101 in adults with moderately severe to severe hemophilia B.

Pathway to Cures, the venture philanthropy fund of the National Bleeding Disorders Foundation, is delighted to name Sylvia Fong, PhD as a member of its Scientific Advisory Group. Dr.

The National Bleeding Disorders Foundation’s (NBDF) National Research Blueprint (NRB), launched in 2020, has a new name: the Bleeding Disorders Research Collaborative.  

Newly published findings suggest positive results from a late-stage clinical trial on a much-needed treatment option for young people with persistent or chronic primary immune thrombocytopenia (ITP).

In early 2021, a pair of much needed evidence-based guidelines on the diagnoses and management of von Willebrand disease (VWD) were issued by an international group of organizations eager to build consensus on VWD screening and improve the care of VWD patients globally.