The National Bleeding Disorders Foundation (NBDF) is proud to announce that Maria E. Santaella, PhD(c), MSN, RN-BC, CPHON, senior vice president of research strategy, has been named editor in chief of Bleeding Disorders Care & Practice.

The National Bleeding Disorders Foundation, a leading U.S. organization dedicated to improving care for people with bleeding disorders, has announced its 2025 NBDF-Takeda Clinical Fellowship Awardees: Deeksha Katoch, MBBS, and Peter Zhao, MD.

Federal: 

Strong Support for FY27 Funding for Federal Bleeding Disorders Programs 

The National Bleeding Disorders Foundation (NBDF) is alerting the community to updated information from Takeda Pharmaceutical Company regarding the discontinuation timelines for HEMOFIL® M and RECOMBINATE®, two therapies used to treat hemophilia A, first announced last year.

The Neil Frick Resources Center (NFRC) is excited to announce new 2026 scholarship opportunities available to the bleeding disorders community.

CSL Behring, the biopharmaceutical company behind the hemophilia B gene therapy HEMGENIX ® (etranacogene dezaparvovec-drlb), announced on March 17, 2026, a temporary global stockout of the therapy. This disruption means that some individuals in countries where HEMGENIX is commercially available may experience delays in accessing treatment.

The National Bleeding Disorders Foundation (NBDF), a leading nonprofit supporting people with bleeding disorders, is proud to congratulate Matthew Delaney, government relations manager, on being named to City & State New York’s 2026 Albany 40 Under 40, an annual list recognizing rising stars in New York State government, politics, and public service.

A Personal Connection to the Bleeding Disorders Community

Federal:  

NBDF Wraps Up Another Successful Washington Days

The National Bleeding Disorders Foundation (NBDF) is pleased to announce a new matching grant partnership with Save One Life that will double the community’s impact for children living with bleeding disorders, including hemophilia A, hemophilia B, and von Willebrand disease in developing countries.

Pathway to Cures (P2C), the venture philanthropy fund of the National Bleeding Disorders Foundation (NBDF), is pleased to announce the appointment of Michael Recht, MD, PhD, MBA, as chief medical and scientific advisor.

BioMarin Pharmaceutical Inc. has announced that it has decided to pull the hemophilia A gene therapy ROCTAVIAN (valoctocogene roxaparvovec-rvox) from the United States market.

The National Bleeding Disorders Foundation (NBDF) mourns the passing of Lynn M. Malec, MD, MSc, a physician, researchers, and champion of the bleeding disorders community.

FY 2026 Funding Update  

Last week, the House of Representatives passed the final fiscal year (FY) 2026 appropriations bills. This includes the FY26 Labor, Health and Human Services appropriations bill, which provides funding for the federal health agencies. There were big wins in the bill for the bleeding disorders community, including:  

FY 2026 Funding Update  

Last week, the House of Representatives passed the final fiscal year (FY) 2026 appropriations bills. This includes the FY26 Labor, Health and Human Services appropriations bill, which provides funding for the federal health agencies. There were big wins in the bill for the bleeding disorders community, including:  

The National Bleeding Disorders Foundation (NBDF) is launching Educate to Elevate, a multi-year initiative to raise public awareness, educate communities, and improve healthcare access for individuals with bleeding disorders. 

The campaign features celebrity advocates including Melora Hardin, Amy Jo Johnson, Jonathan Frakes, Jason Ritter, and Greg Grunberg, alongside medical experts and people living with bleeding disorders, amplifying the experiences and challenges faced by people who live with these disorders every day.

Why This Matters

The National Bleeding Disorders Foundation (NBDF) offers multiple funding opportunities for bleeding disorders researchers across all career stages and disciplines. Through the Judith Graham Pool Postdoctoral Research Fellowships, the Sanofi-sponsored Career Development Award, the Takeda-sponsored Clinical Fellowship, and the Excellence Fellowships, NBDF’s grant programs support diverse research needs, including basic science, clinical projects, and innovative technologies.

After decades of research during which gene therapy was considered a distant goal, FDA-approved gene therapy options for hemophilia are now available in the United States. As these therapies enter real-world clinical practice, patients and care teams are navigating new access and coverage considerations that differ from more traditional treatment models.

The National Bleeding Disorders Foundation (NBDF) has announced the recipients of its 2025 research grant cycle, providing new research funding through its the Judith Graham Pool Postdoctoral Research Fellowship,  Excellence Fellowships in Social Work, Nursing, and Physical Therapy, and the Sanofi-sponsored Career Development Award. This year’s cohort reflects the full breath of bleeding disorders research, spanning basic science, clinical investigation, and qualitative studies led by nursing, social work, and physical therapy professionals.

Federal Updates: 

House of Representatives Reintroduces HELP Copays Act 

The National Bleeding Disorders Foundation (NBDF) has joined with World Federation of Hemophilia (WFH) with issuing a joint statement on a severe adverse event with marstacimab rebalancing agent for hemophilia (Hympavzi®). NBDF believes it is important for the community to be informed as quickly and thoroughly as possible about all adverse events that can impact people’s informed decision-making regarding their treatment. As Pfizer shares more information regarding their ongoing investigation into this event, NBDF will continue to update the community.

The National Bleeding Disorders Foundation (NBDF) is delighted to announce that Luke Luckey of Manchester, Michigan, was selected as the recipient of the 2025 Kevin Child Scholarship (KCS). The Child family selected Luke as this year’s KCS award recipient from among more than 20 applicants.

Hemab Therapeutics recently announced positive results from a Phase 2 clinical study of their investigational subcutaneous therapy sutacimig. 

Sutacimig is a laboratory-engineered bispecific antibody being developed as the first-ever prophylactic treatment for individuals with Glanzmann thrombasthenia (GT), one of several diseases the company is looking to ultimately target with this investigational, sub-Q therapy. 

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