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.


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.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to TRM-201 (rofecoxib), a class of COX-2 selective non-steroidal anti-inflammatory drug (NSAID) for the treatment of hemophilic arthropathy. Rofecoxib is manufactured by Cambridge, Massachusetts-based Tremeau Pharmaceuticals. A start-up company, Tremeau is focused on developing nonopioid pain treatments for rare diseases.

FDA Approves SubQ Treatment for Hemophilia A with Inhibitors

The U.S. Food and Drug Administration (FDA) has approved HEMLIBRA® (emicizumab) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors.

Call to Action on Senate Tax Plan

Call your Senators now and share how the tax reform proposal will harm the bleeding disorders community.

THSNA Summit Coming in March 2018

Learn about the most current clinical and scientific issues related to hemostasis and thrombosis.

In a recent press release, Alnylam Pharmaceuticals announced that the company had achieved “alignment” with the U.S. Food and Drug Administration (FDA) on safety measures and risk mitigation strategies with an eye towards resuming clinical trials of the investigational therapy fitusiran. Alnylam is developing the therapy to treat patients with hemophilia A and B, both with and without inhibitors.

The history of hemophilia treatment is marked by an evolution that has ultimately led to increased life expectancy for persons with hemophilia. By 2050, 20% of the hemophilia population will be over 60 years of age. The increase in life expectancy among persons with hemophilia has been accompanied by the emergence of conditions related to aging in the hemophilia population.

The National Hemophilia Foundation (NHF) is pleased to announce the 2017 Judith Graham Pool (JGP) Postdoctoral Research Fellowship award recipients: Esther Cooke, PhD, from the University of California San Diego, Satish Nandakumar, PhD of Boston Children’s Hospital, and Kasturi Pal, PhD, from The Scripps Research Institute in La Jolla, California.

New Educational Webinars for Healthcare Providers Outside Hematology

Primary care physicians, obstetrician/gynecologists, nurse/nurse practitioners, dentists, oral surgeons and other allied professionals are encouraged to take advantage of these excellent, accredited learning opportunities

The National Hemophilia Foundation (NHF) is pleased to announce Improving Outcomes for von Willebrand Disease and Other Bleeding Disorders in Non-Hematology Health Care Settings, a brand-new series of webinars designed to better educate healthcare providers who do not work in the field of hematology. The project is supported by independent educational grants from Shire and the Centers for Disease Control and Prevention.

 

Interim study results recently published online in the journal Haemophilia, showed that prophylactic use of ELOCTATE® led to joint health improvements in a group of hemophilia A patients. Manufactured by Bioverativ Inc., ELOCTATE®, is a recombinant factor VIII Fc fusion protein (rFVIIIFc) therapy designed to keep the infused clotting factor circulating in the body longer, stretching the time between infusions.

The U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) submitted by Bayer for BAY 94-9027, the company’s long-acting site-specific PEGylated recombinant factor VIII therapy for the treatment of hemophilia in adolescents and adults 12 years of age and over.

The U.S. Food and Drug Administration (FDA) recently granted “breakthrough” therapy designation for valoctocogene roxaparvovec (formerly BMN 270), an investigational hemophilia A gene therapy candidate developed by BioMarin Pharmaceuticals.

The Partners in Bleeding Disorders Education Program recently launched “Clinical Management of Inhibitors Module 3: Immune Tolerance Induction (ITI),” the purpose of which is to sharpen clinical knowledge and skills relating to the nursing role in eradication of inhibitory antibodies to factor VIII and IX via ITI therapy.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Shire’s SHP654, an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A. The therapy was formerly known as BAX 888 – Shire changed the name to SHP654 when it acquired Baxalta in June 2016.

Joint Endorsement of Alexander-Murray

Four leading bleeding disorders organizations joint together to endorse the bipartisan Alexander-Murray Market Stabilization bill.

UniQure recently announced plans to “expeditiously advance” its investigational hemophilia B gene therapy AMT-061 into a pivotal study in 2018. The announcement followed meetings the company had with both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency.

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.