News


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.


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