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Be sure to check back regularly to get our latest news updates.

Study Finds New Information on FVIII Inhibitors

Researchers have made an important discovery that could change the way doctors understand and treat inhibitor development in hemophilia A

Actions from the President adds uncertainty to the health insurnace market.

The National Hemophilia Foundation is pleased to announce that Michael Kuklish of Lillian, Alabama, is the recipient of the 2017 Kevin Child Scholarship. 

Honoring George McCoy

A celebration of life for George McCoy will be held in Cary, North Carolina, on November 12th.

New Educational Activity for Hemophilia Providers Available

This activity discusses the latest advances in the management of hemophilia.

NHF is working with the HTC in Puerto Rico to coordinate aid to people with bleeding disorders.

San Francisco-based Catalyst Biosciences, Inc. recently announced positive clinical data from the first group of patients participating in an ongoing Phase 1/2 proof-of-concept study in individuals with severe hemophilia B. The trial, which is being conducted at three centers in South Korea, is designed to evaluate Catalyst’s investigative, subcutaneous factor IX (FIX) therapy known as CB 2679d. Catalyst develops its therapies using engineered proteases, enzymes that increase the rate of chemical reactions within cells.

NHF Research Recognized by US Specialty Pharmacists

The findings showed the value of collaboration between HTCs, insurers, and speciality pharmacies.

The mission of the Patient Reported Outcomes, Burdens, and Experiences (PROBE) study is to investigate and directly probe patient perspectives on outcomes they deem relevant to their life and care. PROBE aims to develop a new global tool to enhance the direct patient-voice in health care decision-making. The study has partners in both developed and developing countries.

Members of the National Hemophilia Foundation’s (NHF) Medical and Scientific Advisory Council (MASAC) recently gathered during NHF’s 69th Annual Meeting on August 26, 2017 in Chicago. The meeting generated three new documents related to treatment, therapies, women with bleeding disorders, carriers and emergency room management. These documents, which are listed below, were subsequently adopted by NHF’s Board of Directors on September 17, 2017.

Your Access to Healthcare is at Risk!

Call your Senator and ask them to vote NO on the Graham-Cassidy bill, which would jeopardize access to healthcare for people with bleeding disorders.

The Partners in Bleeding Disorders Education Program recently launched a new online learning activity entitled, “Bacteremia: The Good, The Bad, and The Ugly.”

Renowned Hematologist Harold R. Roberts Dies at Age 87

September 9, 2017 saw the passing of Harold R. Roberts, MD at the age of 87. Over the course of his more than 50-year career, the North Carolina native was a highly regarded physician, teacher, researcher, author and mentor in the field of hematology.

Researchers at the Boston Children’s Program in Cellular and Molecular Medicine and the Harvard Medical School recently made important discoveries relevant to the functioning of von Willebrand factor (VWF). The study, “Flow-induced Elongation of von Willebrand Factor Precedes Tension-Dependent Activation,” was published online, August 23, 2017, in the journal Nature Communications.

The U.S. Food and Drug Administration has accepted the Biologics License Application (BLA) and granted priority review for emicizumab, as a once-weekly treatment for adults, adolescents and children with hemophilia A with factor VIII inhibitors. Emicizumab, which is being co-developed by Chugai, Roche and Genentech, is unlike existing treatments in that it is administered subcutaneously via an injection just under the skin, instead of intravenously.

Emergency Preparedness

7 Ways You Can Prepare for Emergencies. Tips and resources that may help you during this hurricane season.

Alnylam Reports Patient Death in Fitusiran Clinical Study

Alnylam Pharmaceuticals, Inc. has reported on the death of a hemophilia A patient who had been participating in a Phase 2 study for the company’s investigational therapy fitusiran.

The Partners in Bleeding Disorders Education Program recently launched a new online learning activity entitled, “Genetics of Hemophilia.” The target audience for this presentation includes nurses and other health care professionals who are part of a U.S. hemophilia treatment center (HTC), who are interested in learning more about the genetic aspects of the condition.

Bayer Submits Application for Long-Acting FVIII Candidate

Bayer applied for a Biologics License Application for BAY 94-9027, the company’s long-acting site-specific PEGylated recombinant factor VIII therapy for the treatment of hemophilia A.

Sangamo Therapeutics, Inc. and Pfizer Inc. recently announced that the first patient received treatment in a phase 1/2 clinical trial designed to evaluate SB-525, the company’s gene therapy program for people with hemophilia A, or factor VIII (FVIII) deficiency. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer for the development and commercialization of hemophilia A gene therapy programs.

The U.S. Food and Drug Administration has approved additional vial sizes for Nuwiq®, Octapharma’s recombinant FVIII therapy to treat hemophilia A patients. The new vial sizes will include 2500, 3000 and 4000 International Units (IU) and will be available in September 2017. The existing sizes include 250, 500, 1000 and 2000 IU.

Disaster Relief for Our Families

Our families in the Houston area are in need of help and here is how you can assist.

Harvey Disaster Relief for Our Families

Our families in the Houston area are in need of help and here is how you can assist.

Irma Disaster Relief for Our Families

Our families in the Florida area are in need of help and here is how you can assist.

Assistance during Hurricane Harvey

Our hearts go out to our families and staff who have been impacted by Hurricane Harvey.  Please know that we are here to help in any way we can.

Pfizer to Build Gene Therapy Facility in North Carolina

Specifically, Pfizer will expand an 11,000-square-foot plant in Sanford, North Carolina that it acquired last year when it bought Bamboo Therapeutics, a biotech company that specializes in gene therapies for certain rare diseases.

FDA Approves New Combination Therapy for all Major Hepatitis C Genotypes

The U.S. Food and Drug Administration (FDA) has approved Mavyret (glecaprevir/pibrentasvir), a combination drug regimen for the treatment of patients with all major genotypes (1-6) of the chronic hepatitis C virus (HCV).

Leading Bleeding Disorders Advocacy Organizations File Hemophilia Discrimination Complaint Against Wellmark Blue Cross and Blue Shield

Complaint argues Wellmark’s withdrawal from the Iowa marketplace discriminates against protected health conditions under the ACA, including hemophilia, and its disclosure of a patient’s personal health information violates HIPAA.

Spark Reports Preliminary Data from Hemophilia A Gene Therapy Trial

Spark Therapeutics, Inc. recently released preliminary data from a phase 1/2 dose-escalation clinical trial for SPK-8011, the company’s investigational gene therapy candidate for hemophilia A.

“Optimizing Transition to Extended Half-Life Factor Replacement Products,” is the latest educational opportunity to become available from Medscape. It is intended for hematologists, pediatricians, nurses, and other healthcare professionals who manage patients with hemophilia.

The activity is part of the online series, “Clinical Advances in Hemophilia: Management for Life,” whose purpose is to increase the knowledge, skills and competence of clinicians in providing individualized management of hemophilia.

FDA Expands Labeling for Epclusa® to Include Co-Infected Patients with HCV/HIV and Warning for HBV Reactivation

The U.S. Food and Drug Administration (FDA) has approved expanded labeling for Epclusa®, a combination therapy for adults with chronic hepatitis C viral (HCV) infection.

The Partners in Bleeding Disorders Education Program recently launched a new online learning activity entitled, “An Overview of the Background, Inheritance and Physiology of von Willebrand Disease.”

The new module is designed to enable the learner to develop an enhanced understanding of the nature of von Willebrand disease (VWD) by providing information about the discovery and evolution of knowledge about the disease. The module also includes an explanation of the production and functions of von Willebrand factor and the varied classifications of VWD.

UniQure N.V. has announced that is has “scaled up” the manufacturing process at its large facility in Lexington, MA. According to a uniQure press release, the facility is designed to manufacture the company’s proprietary hemophilia B gene therapy candidate, AMT-060.

BioMarin Pharmaceutical, Inc. recently provided updated interim results from a phase 1/2 clinical study of BMN 270, the company’s investigational hemophilia A gene therapy. BMN 270 uses adeno-associated viruses (AAVs) as delivery vehicles, or vectors, to carry the genetic codes that prompt the production of the factor VIII (FVIII) protein that is deficient in people with hemophilia A. These AAVs are designed to deliver genetic material into living cells to sustain therapeutic effect without causing disease or triggering significant immune responses.

The Committee of Ten Thousand has requested that NHF make the public aware that their board has chosen not to participate nor support any fundraising efforts for the hemophilia memorial project, led by the National AIDS Memorial Grove.

Choose the Inscription on the Hemophilia Memorial

Vote today for the inscription that will be engraved on the Hemophilia Memorial in San Francisco, CA.

The program will allow bleeding disorders camp staff learn from one another and share best practices.

The Partners in Bleeding Disorders Education Program recently launched an updated version of “Dear Diary: The Importance of Infusion Logs in Home Infusion Therapy.” This activity aims to enable the learner to apply knowledge of the benefits and applications of paper and electronic infusion logs to support their accurate use among hemophilia patients engaged in home infusion.

Alnylam Pharmaceuticals, Inc. and Sanofi Genzyme announced positive results from an ongoing Phase 2 open label extension (OLE) study with fitusiran in patients with hemophilia A and B, both with or without inhibitors. Details from the study were presented at 26th International Society on Thrombosis and Haemostasis Meeting, July 8-13, 2017, in Berlin, Germany.

Shire Submits IND Application for Hemophilia A Gene Therapy Candidate

If effective, the therapy prompts the sustained production of FVIII and enhanced protection from bleeds.

Shire has announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for SHP654, an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A.

Genentech Announces Progress in Two Phase III Emicizumab Studies

Results showed a statistically significant and clinically meaningful reduction in bleed rate, reports states.

Bleeding Disorder Organizations Unite in Opposition to BCRA

NHF, HFA, the Hemophilia Alliance and Coalition for Hemophilia B are united in their opposition to BCRA because of its damaging impact on our community and urge the Senate to reject these harmful policies.

The Senate Bill (BCRA) Will Hurt the Bleeding Disorders Community

Call TODAY and tell your Senator to vote NO on BCRA!


The Partners in Bleeding Disorders Education Program recently relaunched “Understanding Hemophilia,” the purpose of which is to enable the learner to understand the three major steps in normal hemostasis and the basics of treatment and management of patients with hemophilia.

The target audience includes nurses and comprehensive care staff in the U.S. hemophilia treatment center (HTC) network who are responsible for the clinical management and/or education of individuals with hemophilia.

The faculty for this module includes:

Green Park Collaborative, National Hemophilia Foundation, McMaster University, Announce Launch of CoreHEM Project

The collaboration between stakeholders to establish a core set of measurements that would be used to evaluate effectiveness of gene therapies.

The collaboration between stakeholders to establish a core set of measurements that would be used to evaluate effectiveness of gene therapies.

The collaboration between stakeholders to establish a core set of measurements that would be used to evaluate effectiveness of gene therapies.

Novo Nordisk recently announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for REBINYN®, the company’s recombinant, GlycoPEGylated therapy for the treatment of adults and children with hemophilia B (factor IX deficiency). A BLA is a request to the FDA for permission to introduce, or deliver for introduction, a biologic product into interstate commerce.

One of Shire’s investigational therapies, known as SHP656, failed to show significant efficacy in a phase I/II clinical study. The therapy was being developed as an extended half-life treatment for hemophilia A using proprietary technology from Xenetic Biosciences.