PO Box 4214
Morgantown, West Virginia 26504
Phone 304-212-2616

News

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.


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).

 

Keeping Your Informed 

Keeping Your Informed 

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.

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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 

Federal:

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.

PO Box 4214
Morgantown, West Virginia 26504
Phone 304-212-2616

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