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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.
The National Bleeding Disorders Foundation (NBDF) marked its 75th Anniversary in 2023 by recognizing the contribution of various community stakeholders. As part of this campaign, a video was created to celebrate the pivotal role of nursing in comprehensive care for bleeding disorder patients. The video features interviews with experienced nurses who for decades have cared for patients at hemophilia treatment centers throughout the U.S.
The Partners Physician Academy (PPA) is offering a new training course for early career physicians practicing in hematology and specializing in bleeding disorders care.
The training will cover the key conditions that physicians treat and manage when working within the hemophilia treatment center network, such as hemophilia, von Willebrand disease (VWD), and rare bleeding disorders.
Happy Bleeding Disorders Awareness Month! In keeping with the spirit of the month, NBDF’s HANDI Resource Center is pleased to announce the availability of a new Hemophilia Fact Sheet. This two-page PDF document was created to fill the need for a current, accessible resource containing basic information about hemophilia.
Government Relations Update – February 2024
A new study published in the journal Blood Coagulation & Fibrinolysis, looked at the impact of hereditary factor X deficiency (HFXD) on patients and caregivers in the United States. HFXD is an ultra-rare bleeding disorder that occurs in approximately 1 in 500,000 to 1 in a million people globally.
We are pleased to announce that the Emergency Care for Patients with von Willebrand Disease treatment manual is now available in a Spanish language version. Funding for the translation, Cuidado de Emergencia a Pacientes con Enfermedad de von Willebrand, was provided by Akron Children’s Hospital.
NBDF employees are talented, dynamic, and passionate individuals dedicated to supporting the inheritable blood and bleeding disorders community. They value diversity, accountability, service, respect, and support.
Phil Gattone, M.Ed., NBDF President and CEO :
The U.S. Food and Drug Administration (FDA) has approved Fabhalta® (iptacopan), the first oral therapy for the treatment of adults with a rare blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH). It is manufactured by Novartis.
PNH is an acquired (not inherited) condition that causes the immune system to attack and prematurely breakdown red blood cells and platelets. Left untreated, the condition can become serious and even be life threatening. While it can occur at any age, it is most often diagnosed in young adults.
Novo Nordisk recently informed NBDF that they are experiencing a temporary shortage of their product Rebinyn®, specifically in the 3,000 IU vial size. Rebinyn is a 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.
For Immediate Release
CONTACT:
Carrie L. Strehlau
National Bleeding Disorders Foundation
cstrehlau@hemophilia.org
901-230-4665
National Bleeding Disorders Foundation Names Phil Gattone, M.Ed., Next Chief Executive Officer
Gattone brings decades of experience in nonprofit community engagement, fundraising, strategy
Celebrate Black History Month with NBDF!
This year we will share stories from the Black community within the bleeding disorders community. These individuals are truly an inspiration to the entire inheritable blood and bleeding disorders community. We hope that you take the time to read their stores and that their courage is an inspiration to you!
In recent years, the arrival of novel hemophilia treatments that are effective and less burdensome to administer have opened up new possibilities for the consumers of these therapies. This begs the question – How might this evolving landscape be impacting treatment preferences for people with hemophilia?
Takeda announced today that it is conducting a voluntary market withdrawal for two product lots of 650 IU VONVENDI® [von Willebrand factor] in the U.S. Takeda said that the withdrawal is being conducted out of an abundance of caution due to misprinted product labels with the incorrect expiration date.
The expiration date printed on the label on the outside of the package of the affected lots is six months after the actual expiration date (January 27, 2025).
Federal Updates:
Pfizer has announced positive results from their global phase 3 BASIS clinical study of marstacimab, the company’s investigational, subcutaneous therapy that targets an anticoagulant protein known as tissue factor pathway inhibitor (TFPI).
Dear National Hemophilia Foundation Community, Staff and Friends,
When Saylor Behrens' biology professor told her class that hemophilia is the result of incest, she grew angry. She knew better. Her younger brother, Wyatt, has hemophilia A.
It wasn't just the false narrative being spread that made her indignant- it was the sheer lack of awareness about hemophilia that morphed her brother's bleeding disorder into a scary, incredibly rare condition that could be brushed off.
Every day, chapters across the country work with the blood and bleeding disorders community around them. Since 2021, Jacob Murdock has served as Chapter Executive Director in Nevada. He took time out of his busy schedule to tell us why loves the Nevada Chapter.
How long has your Chapter been serving the community?
We were founded as the Hemophilia Foundation of Nevada in 1990. We became a national chapter of NHF in 2011. The chapter was founded by Renee Paper, a nurse and a pioneer in the treatment of women with bleeding disorders.
The Rare Diseases Diversity Coalition (RDDC) is currently seeking applicants for its 2023 Rare Disease Fellowship Program, which is aimed at inspiring the next generation of the medical workforce to work in the rare disease space and achieve greater health equity for people of color living with rare diseases.
The Fellowship allows rare disease organizations to collaborate with fellows to better understand the health impacts of that disease on communities of color. In addition, the Fellow will gain a greater awareness of the challenges of living with rare diseases
NHF’s Medical and Scientific Advisory Council (MASAC) recently issued four new documents, which are now available for easy access.
In July 2020 Ferring Pharmaceuticals suspended manufacturing of the nasal spray STIMATE®, a popular product used to treat certain bleeding disorders.
Between 2013-2017, the “My Life Our Future” (MLOF) project offered eligible individuals with hemophilia free genotyping, which is historically hard to access, expensive, and not covered by insurance.
