If you want to be in the know about what’s going on at our organization, you’ve come to the right place!
-
Keep up to date with the chapter and look back on previous events.
-
Find resources and hear stories from community members in this newsletter just for for women, girls, and people with the potential to menstruate.
Be sure to check back regularly to get our latest news updates.
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.
On April 2, 2024, 8 News Now covered our Las Vegas Chapter's Winter Wine Walk with Stephanie Dupree.
On April 2, 2024, 8 News Now covered our Las Vegas Chapter's Winter Wine Walk with Stephanie Dupree.
A new review published in the European Journal of Haematology describes the challenges inherent in managing both bleeding and excessive clot risk in patients with primary immune thrombocytopenia (ITP).
A new review published in the European Journal of Haematology describes the challenges inherent in managing both bleeding and excessive clot risk in patients with primary immune thrombocytopenia (ITP).
For Immediate Release
CONTACT:
For Immediate Release
CONTACT:
Financing a higher education poses considerable challenges, especially as yearly tuition and other school-related costs continue to rise. For many individuals and families considering a higher education, awareness of any opportunities to defray those costs is critical. In addition, because decisions about school can have both near-term and long-term implications, it is also helpful to understand the many financial variables.
Financing a higher education poses considerable challenges, especially as yearly tuition and other school-related costs continue to rise. For many individuals and families considering a higher education, awareness of any opportunities to defray those costs is critical. In addition, because decisions about school can have both near-term and long-term implications, it is also helpful to understand the many financial variables.
U.S. Hemophilia Treatment Centers (HTCs) are once again participating in the National HTC Patient Satisfaction Survey (PSS). It is launched every three years to help glean valuable feedback from HTC patients and caregivers, with the overarching goal of enhancing care. Participants in the PSS often include people with hemophilia, von Willebrand disease, and other inherited bleeding disorders.
U.S. Hemophilia Treatment Centers (HTCs) are once again participating in the National HTC Patient Satisfaction Survey (PSS). It is launched every three years to help glean valuable feedback from HTC patients and caregivers, with the overarching goal of enhancing care. Participants in the PSS often include people with hemophilia, von Willebrand disease, and other inherited bleeding disorders.
A new paper published in The Lancet Haematology journal includes data from the HOPE-B clinical trial program that is investigating the hemophilia B gene therapy etranacogene dezaparvovec – the product’s commercial name is Hemgenix® (CSL Behring).
A new paper published in The Lancet Haematology journal includes data from the HOPE-B clinical trial program that is investigating the hemophilia B gene therapy etranacogene dezaparvovec – the product’s commercial name is Hemgenix® (CSL Behring).
The HANDI Resources Center Team is pleased to announce new 2024 scholarship opportunities available for the bleeding disorders community!
The HANDI Resources Center Team is pleased to announce new 2024 scholarship opportunities available for the bleeding disorders community!
Government Relations Update – March 2024
Federal: NBDF’s 2024 Washington Days was held March 6-8. NBDF is grateful to the more than 400 volunteer advocates from forty-four states who participated in the 231 meetings with Members of Congress and the state advocacy training. This year’s priority issues are the HELP Copays Act (HR 830/S 1375), appropriations for NIH and CDC programs, and federal action addressing commercial health plans’ narrow prescription drug formularies and exclusions.
State
NBDF recently learned that Novo Nordisk now has 3,000 IU vial size of Rebinyn® back in stock. Rebinyn is the company's recombinant factor IX extended half-life therapy for the treatment of hemophilia B. It is indicated for the on-demand treatment and control of bleeding episodes and the management of bleeding around surgery. It is also indicated for routine prophylaxis to reduce the frequency of bleeding episodes.
The U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application for IXINITY® (Medexus), a recombinant factor replacement therapy to treat patients with hemophilia B.
The new approval expands the product indication to include the on-demand, prophylactic, and perioperative treatment of children under 12 years of age. The previous indication was limited to adults and older children more than 12 years of age.
For Immediate Release
CONTACT:
Carrie Strehlau and Kyla Clark
National Bleeding Disorders Foundation
212-382-3773
kclark@hemophilia.org%C2%A0" title="Email for Kyla Clark">kclark@hemophilia.org
National Bleeding Disorders Foundation Celebrates Five Years of Community Voices in Research
NBDF is pleased to announce HANDI Highlights, which is designed to connect the bleeding disorders community with timely, practical, and readily-accessible resources.
Takeda recently announced positive results from a phase 2 clinical trial of their investigational therapy (Mezagitamab), to treat patients with persistent or chronic primary immune thrombocytopenia (ITP). The therapy is developed with laboratory engineered monoclonal antibodies, which are cloned versions of human antibodies produced by the body’s immune system.
