News

Gene Therapy Struggles in Market

Gene Therapy, 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….

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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]…

Read More

Patient Receives First Hemophilia A Gene Therapy Outside a Clinical Trial

Gene Therapy, 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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

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…

Read More

Gene Therapy Preparedness – It’s Time to Get Educated

Industry News & Research, Living with a Bleeding Disorder

With the advent of gene therapy, patients will need to educate themselves more about eligibility, risks, compliance and costs. And they should educate…

Read More