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NBDF is delighted to dedicate this edition of HANDI Highlights* to the upcoming Rare Disease Day® (RDD), which is observed every year on February 28th (or 29th in leap years) all around the world.
? Tune In! NBDF's Bill Robie Talks HB 1216 & Rx Costs
Today, we're sharing a powerful letter from Emily Ouellette, Executive Director of the Bleeding Disorders Alliance of North Dakota (BDAND), featured on February 18, 2025, in the Minot Daily News.
The U.S. Food and Drug Administration (FDA) recently approved Journavx™ (suzetrigine) 50 milligram oral tablets, a first-in-class non-opioid analgesic developed to treat moderate to severe acute pain in adults. It works by targeting a pain-signaling pathway involving sodium channels in the peripheral nervous system, before pain signals reach the brain. The drug is manufactured by Vertex Pharmaceuticals Inc, which has its North American headquarters in Boston, Massachusetts.
NBDF wants to reaffirm our unwavering commitment to our entire bleeding disorders community. Our mission—enabling every person and family affected by bleeding disorders to thrive—remains unchanged. We stand resolute in our dedication to strengthening equitable healthcare access across our diverse nation, ensuring no one faces their bleeding disorder journey alone.
The National Bleeding Disorders Foundation (NBDF) is excited to announce a new online, enduring, and accredited educational activity available to healthcare providers. The intended audience are hematologists, emergency medicine physicians, nurses, nurse practitioners, physician assistants, pharmacists, and other members of the hemophilia care team in the United States
Our hearts are with everyone affected by the devastating wildfires sweeping through Greater Los Angeles. The National Bleeding Disorders Foundation (NBDF) is actively working with local chapter leaders to support our community during this crisis. We have compiled vital emergency resources, including chapter contacts, county services, transportation assistance, and shelter information. This resource list will be updated regularly as new information becomes available.
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the investigational von Willebrand disease (VWD) therapy VGA039. The therapy is being developed by Star Therapeutics, a biotechnology company based in San Francisco, CA.
The National Bleeding Disorders Foundation (NBDF) is pleased to announce the appointment of three new distinguished members to its Board of Directors, effective January 1, 2025. Additionally, we are delighted to announce the re-election of Susan Hartmann for another term.
The National Bleeding Disorders Foundation (NBDF) is proud to announce its achievement of the prestigious Platinum Seal of Transparency from Candid (formerly known as Guidestar), placing NBDF among the top 0.1% of U.S.
The National Bleeding Disorders Foundation (NBDF) proudly announces that our former President and CEO, Dr. Leonard A. Valentino, has been appointed President of the World Federation of Hemophilia USA (WFH USA). This appointment represents an exciting expansion of Dr. Valentino's dedication to the bleeding disorders community, now on a global scale.
The National Bleeding Disorders Foundation (NBDF) proudly announces that our former President and CEO, Leonard A. Valentino, MD, has been appointed as President of WFH USA. This appointment represents an exciting expansion of Doctor Valentino's dedication to the bleeding disorders community, now on a global scale.
In a significant advancement for von Willebrand Disease (VWD) research and treatment, an international team of experts, including patients, clinicians, and researchers, has developed the first-ever core outcome set (COS) for VWD clinical trials. The initiative, known as coreVWD, establishes standardized outcomes that should be measured in all clinical trials for both prophylaxis and perioperative VWD treatments.
The U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab-mtci) 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.
NBDF Mourns the Loss of Dr. Marion Koerper
The National Bleeding Disorders Foundation is proud to support an important research initiative led by Emily Agen, a genetic counseling graduate student at the University of Michigan, who is investigating our community's understanding of hemophilia gene therapy treatments.
Atlanta, Georgia based Expression Therapeutics recently announced phase 1 clinical trial results based on ET3, the company’s investigational gene therapy. ET3 is developed with novel lentiviral vectors.
Atlanta-based Expression Therapeutics recently announced phase 1 clinical trial results based on ET3, the company’s investigational gene therapy. ET3 is developed with novel lentiviral vectors.
The HANDI team hopes everyone will soon be enjoying all the warmth of the holiday season! To close the year, December HANDI Highlights is dedicated to new and emerging novel technologies for the treatment of people with bleeding disorders.
Sanofi has announced positive results from their pivotal LUNA 3 study, a phase 3 clinical trial of rilzabrutinib, the company’s investigational oral therapy developed to treat persistent or chronic primary immune thrombocytopenia (ITP).
Star Therapeutics, a clinical stage biotechnology company headquartered in San Francisco, CA, recently announced new interim clinical trial data for their investigational von Willebrand disease therapy VGA039. The data comes from VIVID 2, a phase 1 single ascending dose study that is being conducted at multiple sites in the U.S. and abroad.
A new study published in the journal Blood Advances describes a new initiative focused on traditionally underserved and underdiagnosed subgroup within the bleeding disorders community – hemophilia carriers/females with hemophilia.
Federal
2024 Election & What to Expect for the End of the Year
Beginning in 2025 with the 119th Congress and the January 20th Presidential Inauguration, Republicans will take control of the White House and the two chambers of Congress, the Senate flipping from Democratic control and the House remaining Republican. President-elect Trump has already begun shaping his second administration – an effort far more organized than after his first win.
The World Federation of Hemophilia (WFH) is currently seeking feedback from users of the WFH Guidelines for the Management of Hemophilia. The survey includes nine questions focused primarily on users’ experience and preferences, with the goal of making the guidelines as accessible and user-friendly as possible.
The evidence-based, consensus-driven guidelines are developed by international panels of expert healthcare professionals (HCPs) and people with inherited bleeding disorders (IBDs)
In keeping with the spirit of National Family Caregivers Month, November HANDI Highlights is dedicated to resources that help support the caregiving community.
This edition includes resources that speak to challenges universal to all caregivers as well as those unique to caring for individuals with bleeding disorders. Topics include school/daycare coordination, financial planning, caregiver burnout, advocacy, aging, and multi-generational caregiving,
Individuals with severe von Willebrand disease (VWD), the most rare and severe form of the condition, can experience a wide range of bleeding symptoms ranging from relatively mild to serious.
The National Bleeding Disorders Foundation (NBDF) is pleased to announce that three new documents have been issued by the Medical and Scientific Advisory Council (MASAC).
MASAC create recommendations and advisories on treatment, research, and other general health concerns of the inheritable bleeding disorders community.
The National Bleeding Disorders Foundation (NBDF) is pleased to announce that Evan Schow of Bigfork, Montana was selected as the recipient of the 2024 Kevin Child Scholarship (KCS). The Child family selected Evan as this year’s KCS award recipient from among 30 applicants.
October HANDI Highlights is dedicated to an historically underserved group within the inherited bleeding disorders community - women, girls, and people with the propensity to menstruate (WGPPM). While awareness of these populations has increased in recent years, they continue to be underserved in terms of timely diagnosis and management.
On October 11, 2024, Pfizer announced that the U.S. FDA approved HYMPAVZI™ (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric individuals 12 years of age and older with hemophilia A without factor VIII (FVIII) inhibitors, or hemophilia B without factor IX (FIX) inhibitors.
A recent communication has been released regarding the memorial service for the late Neil Frick, a cherished colleague whose passing has deeply affected the National Bleeding Disorders Foundation (NBDF) and the bleeding disorders community.
Neil Frick’s memorial mass is scheduled for Friday, October 25, 2024, at 10:45 AM Eastern time. It will be held at St. Gregory The Great Church, located at 242-20 88th Avenue, Bellerose, New York 11426.
In the wake of Hurricanes Milton and Helene, we at the National Bleeding Disorders Foundation (NBDF) want to take a moment to let you know that we are thinking of you during this difficult time. Our hearts are with all those affected in Florida, Georgia, North Carolina, South Carolina, and Tennessee, where so many are facing overwhelming challenges—especially in accessing vital medical care.
The U.S. Food and Drug Administration (FDA) is holding a public webinar that will speak to the ways in which a strong support network can help mitigate some of the inherent challenges associated with clinical trial participation.
Finding Your Support Team While Participating in a Clinical Trial will feature a panel of patients, care partners, and advocates discussing:
Findings of a new preclinical study published online in Nature Communications may help address lingering questions relevant to the long-term durability of approved hemophilia A gene therapy. The research was led by Lindsey A. George, Director of Clinical in Vivo Gene Therapy and attending physician with the Division of Hematology at the Children’s Hospital of Philadelphia (CHOP).
Celebrating Excellence in the Bleeding Disorders Community
The Georgia World Congress Center in Atlanta buzzed with anticipation as the bleeding disorders community gathered for the pinnacle of the 2024 Bleeding Disorders Conference: The National Bleeding Disorders Foundation's (NBDF) Awards of Excellence ceremony. This night was more than just a distribution of accolades; it was a celebration of resilience, innovation, and unwavering commitment to improving lives.
Honoring a Lifetime of Achievement
The National Bleeding Disorders Foundation (NBDF) is proud to support International Plasma Awareness Week (IPAW), taking place October 7-11 this year. This annual event highlights the critical importance of plasma donation and plasma-derived medicines for our community.
Why It Matters: Plasma-derived therapies are essential for many individuals with bleeding disorders. IPAW helps educate the public about plasma donation and its life-saving impact.
How To Participate
The National Bleeding Disorders Foundation (NBDF) is proud to support Reflections Atlanta, an innovative event aimed at raising awareness about the importance of diversity in clinical trials. The event will take place on October 13th, 2024, in Downtown Atlanta, offering an immersive experience that brings together art, education, and community engagement to spark vital conversations about representation in medical research.
The Capitol Connection session at this year's Bleeding Disorders Conference transformed complex policy into tangible hope for patients. As attendees filled the room, conversations ranged from insurance worries to advocacy strategies, setting the stage for an engaging discussion.
Nathan Schaefer, Senior VP for Public Policy and Access, broadened perspectives on advocacy. "It's about regulatory comments, letters to Congress, and building relationships," he noted, expanding the definition of what it means to be an advocate.
As the sun set on the final day of the Bleeding Disorders Conference (BDC), the atmosphere shifted from professional to playful. The closing night event in the Omni Grand ballroom transformed the conference into a vibrant celebration of community, resilience, and joy.
NBDF’s education team unveiled a game-changing update to its "Steps for Living" initiative at the 2024 Bleeding Disorders Conference, marking a new era in patient education.
Lina Volland, Director of Education at NBDF, highlighted the need for change. "As our resource grew, so did the challenges in keeping it accessible and current," she explained. The old website struggled with inconsistent content and navigation issues.
At this year's Bleeding Disorders Conference, Samantha Carlson and Kristie Harvey-Semi led a session that went beyond our usual discussions of coagulation factors and treatment plans. Instead, we found ourselves exploring the complex history of racial wealth disparities in America.
Kristie, Senior Director of Chapter Advancement and Training at NBDF, guided us through a simulation that brought historical policies to life. With cards representing race, money, and land, she traced the impact of 13 key policies from the post-Civil War era to present day.
