If you want to be in the know about what’s going on at our organization, you’ve come to the right place!

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.