News

Answers to Common Questions Asked about Gene Therapy for Hemophilia

Industry News & Research, Living with a Bleeding Disorder

Did you know? Gene therapy is the use of genetic material to treat or prevent disease. Research into gene therapy for hemophilia…

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Gene Therapy Struggles in Market

Industry News & Research, Living with a Bleeding Disorder

Although it was eagerly anticipated, commercial sales of gene therapy are disappointing. BioMarin’s hemophilia treatment Roctavian had only one US patient last year….

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Learn More About 2024 Bleeding Disorders Scholarships

Industry News & Research, Living with a Bleeding Disorder

The National Bleeding Disorders Foundation (NBDF) HANDI Resources Center Team is pleased to announce new 2024 scholarship opportunities available for the bleeding…

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Hemophilia B Gene Therapy Trial Data Published

Industry News & Research

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…

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Hemophilia B Therapy FDA Approved for Expanded Indication

Industry News & Research

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…

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Takeda Announces Interim Trial Results for Investigational ITP Therapy

Health and Well Being, Industry News & Research

Takeda recently announced positive results from a phase 2 clinical trial of their investigational therapy (Mezagitamab), to treat patients with persistent or…

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Researchers Look at Impact of Factor X Deficiency on Patients/Caregivers

Industry News & Research

Hereditary factor X deficiency is an ultra-rare bleeding disorder that occurs in approximately 1 in 500,000 to 1 in a million people…

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New Hemophilia Fact Sheet Now Available

Industry News & Research, Living with a Bleeding Disorder

The fact sheet includes basic information about hemophilia. The National Bleeding Disorder Foundation’s (NBDF) HANDI Resource Center is pleased to announce the…

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What You Need to Know About Mild Hemophilia

Industry News & Research

Learn why experts consider the term to be a misnomer. by: Donna Behen Look up the word “mild” in the dictionary, and…

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Bleeding Disorders Awareness Month

Advocacy & Legislation, GLHF News, Health and Well Being, Industry News & Research, Living with a Bleeding Disorder

Each March, the community calls attention to inheritable blood and bleeding disorders during Bleeding Disorders Awareness Month. This time gives patients and…

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Von Willebrand Emergency Care Manual Now Available in Spanish

Health and Well Being, Industry News & Research

NBDF is pleased to announce that the Emergency Care for Patients with von Willebrand Disease treatment manual is now available in a Spanish language…

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Researchers Look at Impact of Factor X Deficiency on Patients/Caregivers

GLHF News, Industry News & Research

A new study published in the journal Blood Coagulation & Fibrinolysis, looked at the impact of hereditary factor X deficiency (HFXD) on patients…

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Respiratory Viruses are Increasing this Winter – Protect Yourself from the Seasonal Spike of COVID-19

COVID-19, Health and Well Being, Industry News & Research

Infections from respiratory viruses, including the latest COVID-19 variants, are increasing this winter—but vaccines offer effective safeguards for people with inheritable blood…

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FDA Approves Therapy for Rare Disorder Affecting Red Blood Cells

Industry News & Research

The U.S. Food and Drug Administration (FDA) has approved Fabhalta® (iptacopan), the first oral therapy for the treatment of adults with a…

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Novo Nordisk Experiencing Shortage of Rebinyn 3,000 IU Vials

Industry News & Research

Novo Nordisk recently informed NBDF that they are experiencing a temporary shortage of their product Rebinyn®, specifically in the 3,000 IU vial…

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Study Suggests Hemophilia Patient Preferences Evolving Along with Treatments

Health and Well Being, Industry News & Research, Living with a Bleeding Disorder

In recent years, the arrival of novel hemophilia treatments that are effective and less burdensome to administer have opened up new possibilities…

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Takeda Issues Voluntary Product Withdrawal

Industry News & Research

Takeda today announced that it is conducting a voluntary market withdrawal for two product lots of 650 IU VONVENDI® [von Willebrand factor]…

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Patient Receives First Hemophilia A Gene Therapy Outside a Clinical Trial

Industry News & Research

Roctavian™ was approved in the summer of 2023 for the treatment of adults with severe hemophilia. The first hemophilia A gene therapy…

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Takeda’s Voluntary Withdrawal: Alert on Misprinted Labels for VONVENDI®

Industry News & Research

Takeda announces a voluntary withdrawal for 650 IU VONVENDI® due to misprinted labels showing incorrect expiration dates. Takeda announced today that it…

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Nurses Working in the Bleeding Disorders Community Share Their Stories in These 2023 Video Interviews

Health and Well Being, Industry News & Research

In recognition of the National Bleeding Disorders Foundation’s 75th anniversary, the Nursing Working Group gathered recorded video interviews of established nurses working…

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Who’s at Higher Risk for Iron Deficiency?

Health and Well Being, Industry News & Research, Living with a Bleeding Disorder, Women with Bleeding Disorders

Learn why women, girls, and people with the potential to menstruate (WGPPM) who have hemophilia, von Willebrand disease, or other inheritable blood…

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Yale Researchers Explore the Possibilities of von Willebrand Factor

Industry News & Research, Women with Bleeding Disorders

Researchers at the Yale School of Medicine have been studying the key clotting protein von Willebrand factor (VWF), to shed light on…

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Statement: HFA and NBDF Address WHO Recommendations

Health and Well Being, Industry News & Research

Hemophilia Federation of America (HFA) and the National Bleeding Disorders Foundation (NBDF) are deeply concerned by the World Health Organization’s (WHO) recommendation…

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Pilot Study Explores Educational Needs of HTC Physical Therapists

Industry News & Research, Living with a Bleeding Disorder

Though it is generally recognized in the bleeding disorders community that physical therapists (PTs) are essential members of the core interdisciplinary teams…

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