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.

In a session that felt more like a heartfelt conversation among friends than a formal presentation, the National Bleeding Disorders Foundation (NBDF) research team opened our eyes to a powerful truth: the most valuable experts in bleeding disorders research are often the patients themselves.

A manufacturing deviation at NovoNordisk's Danish facility has led to potential underfilling of vials for NovoSeven©, NovoEight©, and Esperoct© - crucial treatments for hemophilia and other blood disorders. While alternative treatments exist for NovoEight and Esperoct, the NovoSeven shortage could have significant implications due to its unique indications and limited alternatives.

In a powerful session, the "Black & African American Voices" panel brought to light the often-overlooked challenges faced by people of color in the bleeding disorders community. The discussion, featuring a diverse group of speakers, painted a vivid picture of the intersectionality between race, health, and identity.

At the Bleeding Disorders Conference, a pioneering session led by Dawn Rotellini of the National Bleeding Disorders Foundation put women's health in the spotlight. The panel introduced a game-changing concept: WGPPM (Women, Girls, and People with Potential to Menstruate), signaling a shift towards more inclusive care.

At the recent Bleeding Disorders Conference, an interactive pre-conference session on von Willebrand Disease (VWD) offered attendees a tantalizing look into the future of treatment and care. The session, sponsored by CSL Behring and led by Lena Volland, brought together experts, patients, and innovators to discuss groundbreaking initiatives and technologies.

Parents of children with complex medical needs gathered to share their experiences and strategies for managing stress. The session focused on three primary challenges: lack of control, suddenness, and ambiguity.

"It's like trying to catch lightning in a bottle," one mother said, describing her attempts to maintain control in unpredictable situations. From unexpected ER visits to navigating school accommodations, these parents face daily challenges that test their resilience.

In a groundbreaking session at the recent Bleeding Disorders Conference, Dr. Karen Zimowski unveiled the latest advancements in treating inherited bleeding disorders, signaling a new era of personalized care.

Gone are the days of one-size-fits-all approaches. Today's treatment landscape is as diverse as the patients it serves. From traditional factor replacement therapies to cutting-edge gene therapy, options abound. "It's not just about stopping bleeds anymore," Dr. Zimowski explained. "It's about preventing them and improving overall quality of life."

At the recent Bleeding Disorders Conference, a powerful session shed light on the unique challenges faced by teens transitioning to adulthood while managing their condition. The message was clear: with the right tools and support, these young adults are more than capable of thriving.

"It's not just about managing bleeds anymore," shared Shawn B., a post-grad student from Northeastern University. "It's about building a life."

At this year's Bleeding Disorders Conference, a groundbreaking initiative took center stage: the Thrive campaign. Developed in collaboration with the CDC, this nationwide effort aims to empower individuals with bleeding disorders to live life to the fullest.

Dr. Michael White from Emory University and Erica, a parent advocate, introduced the campaign's holistic approach. "Thriving isn't just about managing bleeds," Dr. White emphasized. "It's about addressing the whole person—mind, body, and spirit."

At the recent National Bleeding Disorders Foundation conference in Atlanta, a revolutionary approach to medical research took center stage. The National Research Blueprint (NRB) is set to transform how we understand and treat bleeding disorders, with patients themselves at the helm.

Gone are the days of research confined to sterile labs. The NRB brings the process into the living rooms of those who matter most: the patients. "We're not just subjects anymore," says Sammy Thompson, a conference attendee living with hemophilia. "We're partners in this journey."

In a dynamic meeting that buzzed with enthusiasm, NBDF's President and CEO Phil Gattone captivated chapter representatives with his vibrant vision for the bleeding disorders community. The atmosphere was electric as Phil shared his insights and plans for the future.

With genuine passion, Phil took the audience on a journey through the organization's rich 76-year history, acknowledging the living legends who've shaped our community. "We've come a long way," he remarked warmly, "and we're just getting started."

Hey there, BDC Daily readers! Hold onto your hats, because this year's Bleeding Disorders Conference just showed us that our community leaders aren't just thinking outside the box—they're tossing the box out entirely!

In a night that will be remembered for years to come, the Bleeding Disorders Conference 2024 kicked off with an opening ceremony that seamlessly blended history, hope, and harmonies. As the lights dimmed in the packed hall, attendees found themselves on an emotional journey through the past, present, and future of the bleeding disorders community.

The National Bleeding Disorders Foundation’s (NBDF) 76th Annual Bleeding Disorders Conference (BDC) is finally here, and there’s no better place to host it than in the vibrant and welcoming city of Atlanta, Georgia—the great Peach State. Whether you're here for the first time or a returning attendee, this year promises to be an unforgettable experience filled with learning, community, and inspiration. 

At the Industry Stakeholder Meeting (Public Policy) on September 11, 2024, the National Bleeding Disorders Foundation (NBDF) Public Policy team once again demonstrated the incredible power of collective advocacy in shaping a brighter future for the bleeding disorders community. For those of us living with, or caring for someone with, a bleeding disorder, it’s critical to know that our voices are being heard at the highest levels of government—and this session showed just how much progress is being made on our behalf.

MEDIA CONTACT: 

Kyla Clark
Director of Public Relations and Communications
kclark@bleeding.org
M: 347-920-0047

The National Bleeding Disorders Foundation (NBDF) is pleased to announce a new enduring and accredited educational activity available to healthcare providers. "Exploring Non-Factor Replacement Therapies for Hemophilia Care: Achieving Positive Outcomes," was developed through a strategic collaboration between Medscape and NBDThe National Bleeding Disorders Foundation (NBDF) is pleased to announce a new enduring and accredited educational activity available to healthcare providers.

Back-to-school season is in full swing as summer draws to a close. Across the country, many students have already returned to school while others will begin “hitting the books” after the Labor Day weekend.

The National Bleeding Disorders Foundation (NBDF) is excited to share a new collection of resources about Gene Therapy, especially for those in the Hemophilia community. This collection includes easy-to-understand scientific articles, answers to common questions, and a glossary of key terms.

BioMarin recently announced that they will be changing the scope of their efforts to manufacture and market their gene therapy product ROCTAVIAN.™ The product was first approved in the summer of 2023 for the treatment of adults with severe

NBDF is excited to inform U.S.-based clinicians of new live and virtual educational opportunities from overseas! This coming fall, our friends at the Irish Haemophilia Society (IHS) will be offering a series of medical-based webinars that speak to key topics relevant to the care of patients with inherited bleeding disorders.

As we enter the “dog days” of summer with high temperatures and heat weighing folks down throughout much of the US, it’s easy to become lethargic about one’s health, particularly when it comes to addressing issues of pain. This month, HANDI has chosen to feature several resources on the assessment and treatment of pain. 

Pfizer has announced positive topline results from their phase 3 clinical study of giroctocogene fitelparvovec, the company’s investigational hemophilia A gene therapy. 

Government Relations Update – July 2024

Federal

For Immediate Release 
CONTACT:  
Kyla Clark 
National Bleeding Disorders Foundation 
347-920-0047
kclark@hemophilia.org%C2%A0" title="Email for Kyla Clark">kclark@bleeding.org 

NBDF Unveils THRIVE Campaign: Empowering Individuals with Bleeding Disorders through 6 Essential Steps

Pfizer recently provided new clinical trial updates for their investigational therapy marstacimab. The data was presented last month at the 2024 annual meeting of the European Hematology Association (EHA) in Madrid. Marstacimab is a laboratory-engineered monoclonal antibody developed to treat hemophilia A and B patients, with or without inhibitors.