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

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.

Fundraisers are an important way to give back to your school and raise money to support causes you care about. However, fundraisers aren’t the only way to get involved.

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.

Whether they’re familial, romantic or platonic, relationships are vital to beneficial and stable lives. So how can we learn to build positive and enriching relationships?

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.

This August, we celebrate National Water Quality Month. How can you have an impact on water quality?

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.  

For individuals and their families, the words “You need a new organ” are earth-shattering. Luckily, we can make a difference.

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).

How can you live an altruistic lifestyle when negativity is on your mind? Today we've got the answers as we share six tips to help train your brain to be more positive.

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.

Pfizer recently announced results from a clinical study which is currently evaluating HYMPAVZI™ (marstacimab-hncq) for adults and adolescents living with hemophilia A or B with inhibitors. The therapy was approved by the U.S.

Summer is now in full swing, with the sunny promise of more opportunities to get outside and be active. With that in mind, the Neil Frick Resource Center (NFRC) is highlighting resources to encourage better joint health in people with hemophilia and other bleeding disorders. This edition references a series of multi-formatted resources, including articles, videos, educational publications, and medical recommendations. 

NBDF is delighted to share our latest Collaborative Learning Exchange (CLE), “Assessing the Wellbeing of Women with Bleeding Disorders: Focus on Bone and Mental Health.” Recorded live and virtually on June 25^th, the activity featured illustrative case studies, presentations, and thoughtful discussions about the critical need for regular screening for anxiety/depression and bone health in women with inherited bleeding disorders.

The National Bleeding Disorders Foundation was saddened to learn of the passing of Carol Kasper, MD, a pioneer in the treatment of bleeding disorders, who died on July 8, 2025.

Whether it’s a staycation or a world trip —you deserve a break this year.

When you have an efficient morning routine, you set an effective tone for the rest of the day. Here are five ways to establish a productive morning routine.

Federal:
 

Action Alert on H.R. 1 (Budget Reconciliation): As of July 1, 200 bleeding disorders advocates had taken over 250 300 actions to contact Members of Congress in response to NBDF’s Grassroots Action Alert on the “One Big Beautiful Bill”.  
 

Budget Reconciliation Update
 

Latest news: H.R. 1 passed the Senate and House last week and was signed by the President.
 

This week, Congress passed a bill, the “One Big Beautiful Bill Act,” that will have widespread impacts on the bleeding disorders community – together with millions of others across the country who get health insurance through Medicaid or the Affordable Care Act Marketplaces.

Hemab Therapeutics recently presented clinical and preclinical stage data associated with two of their investigational therapies - Sutacimig (formerly HMB-001) and HMB-002. Hemab is a biotechnology company that specializes in the development of therapies that target underserved bleeding and thrombotic disorders. The data were presented last week at the Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Washington, DC.

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. A newly published paper takes a 200+ year view of this history and provides a better understanding of just how this underserved population has slowly but surely garnered greater visibility and awareness – if not equitable care – over the decades. 

Setting goals is simple. We’ve all heard different phrases, acronyms and ways to properly set a goal for life. However, accomplishing goals can be a whole different task.

Roche (and Roche group member Chugai) recently presented data from a phase I/II clinical study of their investigational bispecific antibody therapy NXT007, in people with hemophilia A who do not have factor VIII inhibitors. NXT007 is being positioned as a next-generation subcutaneous prophylactic treatment that builds on the technology used to develop emicizumab (Hemlibra®). 

Pathway to Cures’ investment in Spark Biomedical is an example of how we foster the development of innovative approaches to healthcare access and unmet medical needs in the blood and bleeding disorders community.

If you’re looking for a way to get outside while also improving your whole self, gardening may be the hobby for you.

The National Bleeding Disorders Foundation (NBDF) is delighted to announce a new accredited education opportunity for health care providers focused on personalized care for individuals with von Willebrand disease (VWD).

“Restoring Control in von Willebrand Disease: Collaborative Approaches for Delivering the Right Treatment for the Right Patient at the Right Time,” is an enduring activity planned and implemented by the PeerView Institute (PVI) for Medical Education and NBDF. 

The U.S. Food and Drug Administration (FDA) has approved the ConcizuTrace™ ELISA Companion Diagnostic (CDx) test, which helps to determine the amount of concizumab-mtci that is circulating in an individual who has taken the product.

We all know volunteering has a positive influence, but did you know that it can actually assist your health?

In a May 27th letter to the hemophilia community, it was confirmed that Spark Therapeutics has been fully integrated into the Roche Group. Spark first became a member of the Roche Group in 2019 with the aim of continuing to advance potential gene therapies for hemophilia and many other genetic diseases.

No matter how old you are, what your musical taste is or whether or not you can read a note, there’s never a wrong time to learn how to play an instrument.