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.

The National Bleeding Disorders Foundation (NBDF) is excited to announce a new accredited educational opportunity for healthcare providers. This live/virtual program will focus on optimizing non-factor replacement therapy for hemophilia patients. 

On Friday, June 28th, Novo Nordisk sent a letter to NBDF sharing more information about the current product issue related to NovoSeven® RT and Novoeight ®. The letter is in response to questions that NBDF asked Novo to better understand the cause of this product issue, the potential impact on patients in the United States, and how Novo plans to address it.  NBDF appreciates this additional information, and we will continue to work together with our global partners to raise additional questions and concerns as needed.   

Government Relations Update – June 2024

Federal

The National Bleeding Disorders Foundation (NBDF) is excited to announce a new accredited educational opportunity for healthcare providers. This live/virtual program will focus on optimizing non-factor replacement therapy for hemophilia patients. 
 

It has been several decades since the publishing of large U.S. studies of neurodevelopment and other cognitive functions in children and young adults with hemophilia. Results of this earlier research that evaluated hemophilia and neurocognitive development suggested that the disease was associated with poorer outcomes compared with the general U.S. These lesser outcomes were observed specifically in areas such as academic achievement, attention, behaviors, and emotional and adaptive functioning. 

The National Bleeding Disorders Foundation (NBDF) is pleased to announce a series of educational videos on clinical issues in women, girls, and people with the propensity to menstruate (WGPPM). They were created for physicians, physician assistants, nurse practitioners, and other clinicians who treat patients with bleeding disorders, including von Willebrand disease (VWD), hemophilia A or hemophilia B. We are sharing this information for those individuals who may have missed our earlier announcement.

The National Bleeding Disorders Foundation (NBDF) is pleased to announce the availability of a series of educational videos on the use of novel non-factor therapies. These videos were designed for clinicians who regularly prescribe therapies for people with hemophilia A and B. We are sharing this information for those individuals who may have missed our earlier announcement.

NBDF has learned about a product issue with NovoSeven^® RT and Novoeight^®. This is not a product recall, instead there is a concern about the manufacturing process. This manufacturing issue has led to a temporary shortage of the NovoSeven^® RT 1 mg and 2 mg vials and some strengths of Novoeight^® at the Novo Nordisk Distribution Center (NNDC).

It’s June and with Summer almost upon us, it’s a time to head outside for more fun in the sun! It’s also a great time to consider a few resources on maintaining healthy joints before engaging in outdoor activities. This edition of HANDI Highlights contains relevant content links about the anatomy and treatment of joint bleeds, safe participation in sports, and important tips on prevention and exercise.

The National Bleeding Disorders Foundation (NBDF) is thrilled to announce the upcoming launch of the revamped Steps for Living website, your ultimate educational resource for living with inheritable blood and bleeding disorders. Whether you're a patient, parent, caregiver, or healthcare provider, this updated site is designed to guide you through every stage of life, helping you and your loved ones live healthier, happier lives.

For Immediate Release 
CONTACT:  

Results of a new study published in the Journal of Blood Medicine (JBM), indicate that people with hemophilia A (HA) who have been on a prophylactic treatment regimen have a reduced risk for intracranial hemorrhage (ICH). 

The U.S. Food and Drug Administration (FDA) has approved updated prescribing information for ALTUVIIIO™ (Sanofi) to include the full results of the phase 3 XTEND-Kids clinical trial. 

For Immediate Release 
CONTACT:  

May is Mental Health Awareness month and HANDI, in keep with this theme, is excited to focus on resources that support mental well-being. This edition highlights this important topic from a variety of perspectives and through multiple formats including videos, articles, and podcasts.

Late in 2023 the U.S. Food and Drug Administration (FDA) approved a pair of new therapies that represented a significant scientific breakthrough in gene therapy to treat patients with sickle cell disease (SCD), a rare condition with historically few viable treatment options. 

For Immediate Release 
CONTACT:  

The U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (Pfizer, Inc.), a new hemophilia B gene therapy product. It is designed with bioengineered adeno-associated virus (AAVs) vectors to introduce a working copy of the factor IX gene. The therapy is administered as a one-time intravenous infusion to stimulate the long-term production of clotting factor IX (FIX) and prevent bleeding. This represents the second hemophilia B gene therapy product to be approved in the U.S.

New documents issued by NBDF’s Medical and Scientific Advisory Council (MASAC) cover a wide range of topics relevant to bleeding disorder patients in the U.S. and abroad. They encompass recommendations for treatment with licensed therapies, chronic pain management and physical therapy, gene therapy terminology, and bleeding disorders diagnosis in women and girls. Also included are two new resolutions challenging international recommendations that could contribute to negative health and quality of life implications for patients.