Newsroom


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.


The designation is for people with hemophilia A without inhibitors.

Dawn Rotellini

NHF is expanding its work with the bleeding disorders community around the world.

ICER is an independent and non-partisan research organization that evaluates the clinical and economic value of prescription drugs, medical tests, and other healthcare and healthcare delivery innovations.

Grifols, S.A. recently announced that in 2017, the company donated over 25 million international units (IU) of factor products used to treat hemophilia A and B. According to a recent press release, the donation is part of Grifols’ commitment to provide a minimum of 200 million IU of factor to the World Federation of Hemophilia (WFH) Humanitarian Aid Program over a span of eight years – Grifols began this commitment in 2014. The company expects to make an additional donation of nearly 25 million IUs in 2018.

NHF CEO Val D. Bias

National Hemophilia Foundation CEO Val D. Bias spoke to the leaders of NHF’s 52-chapter networks at the NHF 2018 Chapter Leadership Seminar in San Francisco, California.

NHF 70th the Fight to Preserve ACA Patient Protections Goes On

2018 marks NHF’s 70th year as the bleeding disorders community’s chief advocate.

Graduation hat

Applications are now open! The selected KCS recipient will receive an award of $1,000 toward their tuition costs.

Updated statement from Genentech on Hemlibra, March 28, 2018.

Rockville, Maryland-based Precision BioLogic is collaborating with Roche and Genentech on the development of a new diagnostic kit for inhibitor testing of individuals with hemophilia A. The modified Nijmegen-Bethesda Assay (MNBA) screening kit was developed for broad utility for both laboratories seeking a standardized inhibitor assay for clinical management and multi-center clinical studies of patients with hemophilia A.

Novo Nordisk Announces BLA Submission for Extended Half-Life FVIII Therapy

NHF received information from Genentech’s Clinical Development Lead regarding reports of the death of patients utilizing Hemlibra (emicizumab).

“Advances in Managing Inhibitors in Patients with Hemophilia A,” is the newest educational opportunity to become available through Medscape. It is intended for hematologists, pediatricians, nurses and physicians specializing in emergency medicine. The goal of the activity is to discuss recent advances in the understanding of inhibitors in patients with hemophilia A, including the risks associated with inhibitors as well as current and new approaches to management.

Upon completion of this activity, participants will have increased knowledge of:

Aptevo Therapeutics recently announced patient-reported data on individuals currently being treated with IXINITY®, the company’s recombinant factor IX product. IXINITY® is indicated for the control and prevention of bleeding episodes and for perioperative management in adults and children, 12 years of age or older, with hemophilia B. The results were presented at the Thrombosis and Hemostasis 2018 Summit of North America, held in on March 8-10, 2018 in San Diego, CA.

CSL Behring Discontinues Production and Distribution of Monoclate-P®

Women with linked arms

The study focused on disparities associated with bleeding symptoms, age at diagnosis and provider interventions for females with bleeding disorders.

Team NHF

Runners in the NYC Half Marathon are an inspiration to the bleeding disorders community.

Novo Nordisk recently announced that they have submitted a Biologics License Applications (BLA) to the U.S. Food and Drug Administration for N8-GP (tuco-tuco alfa pegol), a recombinant, extended half-life factor VIII (FVIII) product intended for the prevention and treatment of bleeding in people with hemophilia A.

a young man at his computer

If you or someone you care for had contact with an HTC in 2017, your feedback is needed!

Ron Bark

NHF adds their condolences to all those whose lives Ron Bark touched.

Barry Haarde

Barry Haarde was a cyclist and community activist whose strength will never be forgotten.

Registration is now open for the National Heart, Lung, and Blood Institute (NHLBI) State of the Science Workshop, Factor VIII Inhibitors: Generating a National Blueprint for Future Research. The goal of the workshop is to solicit hemophilia community-wide input into a coordinated national blueprint for future basic, translational, and clinical research focused on factor VIII immunogenicity and factor VIII inhibitor prevention and eradication.

Investigators at the Children’s Hospital of Los Angeles (CHLA) recently published a retrospective review of patient clinical data, the findings of which reflect a series of largely successful transitions to extended half-life (EHL) therapies. The data was drawn from patients treated at CHLA’s Hemostasis and Thrombosis Center (HTC).

Steven Pipe, MD

Steven Pipe, MD, NHF's Chair of MASAC, looks at the future of bleeding disorders treatment.

Novo Nordisk recently announced the availability of REBINYN®, the company’s recombinant GlycoPEGylated product for the treatment of adults and children with hemophilia B (factor IX deficiency). The therapy was first approved by the U.S. Food and Drug Administration in May of 2017.

REBINYN® is indicated for on-demand treatment and control of bleeding episodes, and the perioperative management of bleeding in adults and children with hemophilia B. It is not indicated for routine prophylaxis or for immune tolerance induction in patients with hemophilia B.

This webinar is free and open to public health professionals, clinicians, and researchers who want more information about hemophilia.

On March 1, 2018, the National Institutes of Health (NIH) will recognize Rare Disease Day®, which is dedicated to raising awareness about rare disorders, the communities affected by them and current research collaborations in relevant scientific fields. According to the NIH, rare diseases affect an estimated 25 million people in the United States. The annual event is sponsored by the National Center for Advancing Translational Sciences (NCATS) and the NIH Clinical Center (NIHCC).

flooded neighborhood

Plan for the unexpected with these emergency preparendness resources.

The National Hemophilia Foundation (NHF) is excited to announce the availability of a new educational webinar created to improve the overall knowledge of physicians in evaluating and screening women and girls with heavy menstrual bleeding for a bleeding disorder. 

hands holding files

In the past few weeks, we have received many inquiries regarding the scope of the injunction Shire is seeking in its lawsuit against Genentech/Roche.

helping hands

The Patient Access Network (PAN) Foundation announces a new patient insurance premium assistance program for individuals with hemophilia.

The Patient Access Network (PAN) Foundation has opened a new patient assistance program for individuals with hemophilia. PAN is an independent, national 501 (c)(3) organization dedicated to helping federally and commercially insured people living with life-threatening, chronic and rare diseases with out-of-pocket costs for their prescribed medications. The program was announced in a PAN press release on January 9, 2018.

The Patient Access Network (PAN) Foundation has opened a new patient insurance premium assistance program for individuals with hemophilia. PAN is an independent, national 501 (c)(3) organization dedicated to helping federally and commercially insured people living with life-threatening, chronic and rare diseases with out-of-pocket costs for their prescribed medications. The program was announced in a PAN press release on January 9, 2018.

NHF CEO Val D. Bias

But it is not our story -- it's yours.

Pipettes

Trial investigators will evaluate MarzAA’s ability to curb spontaneous bleeding episodes in patients.

Medscape recently launched a new education opportunity for healthcare providers. “Hemophilia 2018: Current and Emerging Therapies” is a discussion focused on the latest advances in the management of hemophilia, including the evolving role of extended half-life factor products, emerging data on small molecule approaches and gene therapy. The activity, which centers on a video recording of a live presentation at the 2017 American Society of Hematology Annual Meeting, is intended for hematologists, pediatricians, and other healthcare professionals who treat patients with hemophilia.

Medscape recently launched a new education opportunity for healthcare providers. “Hemophilia 2018: Current and Emerging Therapies” is a discussion focused on the latest advances in the management of hemophilia, including the evolving role of extended half-life factor products, emerging data on small molecule approaches and gene therapy. The activity, which centers on a video recording of a live presentation at the 2017 American Society of Hematology Annual Meeting, is intended for hematologists, pediatricians, and other healthcare professionals who treat patients with hemophilia.

The activity reviews current treatment options for hemophilia A, challenges to optimal treatment, and future research directions.

NHF Statement on Shire Injunction Filing on Roche/Genentech

NHF is aware that recently Shire sought an injunction against Roche/Genentech.

BioMarin Reports Positive Gene Therapy Trial

BioMarin provided a series of updates on their investigational hemophilia A gene therapy candidate valoctocogene roxaparvovec.

Survey for African Americans and Latinos with Hemophilia and VWD Type III

People who are African-American or Latinx and have hemophilia A or B, or VWD type 3 are at an increased risk of developing an inhibitor.

FDA Lifts Hold on Fitusiran Trials

The FDA has lifted a hold on clinical studies involving the investigational therapy fitusiran.

In a recent press release Shire announced that they have entered into a collaboration agreement with Rani Therapeutics. This agreement allows Shire to conduct research on the use of Rani Pill™ technology for the oral delivery of factor VIII (FVIII) therapy for patients with hemophilia A. As part of the agreement, Shire obtains exclusive rights to conduct research on, evaluate, and pursue a worldwide, exclusive license for the development and commercialization of such a therapy.

Spark Therapeutics provided clinical trial updates on the company’s investigational gene therapies for hemophilia A and B, known respectively as SPK-8011 and SPK-9001. The updates were presented at the recent 59th American Society of Hematology (ASH) Annual Meeting and Exposition, which was held December 9-12, 2017 in Atlanta, GA.

NHF Healthcare Update: Congressional To-Do List

Congress has a lengthy to-do list before the end of the year.  NHF is closely monitoring numerous issues affecting the bleeding disorders community including:

Tax Reform

Results in HEMLIBRA® Study for Patients Without Inhibitors

Genentech recently reported results from the phase III HAVEN 3 study, which showed that the prophylactic use of HEMLIBRA® led to significantly reduced bleeds in adult and adolescent patients with hemophilia A, without inhibitors. The therapy was co-developed by Genentech, Chugai and Roche.

It's Not Too Late: Take Action on Tax Reform

 

 

NHF Announces the 2017 NHF/Novo Nordisk Career Development Award Recipient

NHF is pleased to announce Christopher J. Ng, MD, Assistant Professor of Pediatrics, University of Colorado Denver, as the recipient of the 2017 NHF/Novo Nordisk Career Development Award (CDA).

The Partners in Bleeding Disorders Education Program recently launched “Psychosocial Considerations in Bleeding Disorder Care: Birth to Preadolescence.” This online learning activity is focused on the developmental and psychosocial issues unique to children with bleeding disorders, specifically those from infancy through preadolescence. The activity also encompasses the psychosocial considerations that may be seen in families of affected children.     

Action to Hope Study Open to Hemophilia Community

The study seeks to shed light on the barriers people with hemophilia who are obese or overweight have to losing weight.

The National Hemophilia Foundation (NHF) would like to make healthcare providers aware of three monographs that are available through NHF’s resource center HANDI. Each booklet is devoted to a key subject in bleeding disorders management, including the genetics of hemophilia, von Willebrand disease and inhibitors in hemophilia.

Swedish Orphan Biovitrum (Sobi) recently announced the successful enrollment of the first hemophilia A patient in the company’s ReITIrate clinical trial. The phase 4 study, which is being sponsored by Sobi and Bioverativ Therapeutics, is designed to evaluate the use Eloctate™ for immune tolerance induction (ITI) therapy in hemophilia A patient with inhibitors. ITI is an effective approach to inhibitor eradication in which the body begins to tolerate a therapy after daily doses of factor are administered over time.