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.

This piece is a joint effort by Amy Niles, executive vice president at the PAN Foundation, and Kollet Koulianos, vice president of payer relations at the National Hemophilia Foundation.


Each year on January 1, health plans reset for millions of Americans with serious and complex illnesses, restarting the slow and often expensive process of working toward their annual deductible and maximum out-of-pocket limit. But for people with co-pay accumulator policies built into their health insurance plans, there’s a much steeper hill to climb.

BioMarin recently shared a community update on their Hemophilia A Clinical Development Program for valoctocogene roxaparvovec, the company’s investigational gene therapy.

WASHINGTON, D.C.— December 31, 2021 — In response to additional rulemaking for the Centers for Medicare & Medicaid Services’ (CMS)


Media & Community Inquiries

Brett Spitale, VP of Advancement

Dec. 31, 2021 – New York, NY – NHF is deeply saddened to announce that former NHF CEO and community member, Val Bias, passed away suddenly on Thursday, Dec. 30.

Val and I first got to know one another 30 years ago, when we were opposing one another in an election for president of the National Hemophilia Foundation (US). I was fighting for HIV/AIDS advocacy from within, from the Board, and Val was part of a more activist group that was impatient the NHF wasn’t doing enough, fast enough. He and I came together, realized our goals were the same, and also recognized we were so much stronger together.

Results of a recently published study suggest that intense physical activities, particularly those that include a high collision-risk, can increase bleeding risk in individuals with severe or moderately severe hemophilia A. Investigators also found that increasing the time between the administration of factor (FVIII) and physical activity also heightened bleeding risk.

Sigilon Therapeutics recently announced a “strategic reprioritization,” as the company shifts focus to their non-hemophilia, investigational therapies, including those for MPS-1 and diabetes. This closely follows recent setbacks associated with their phase 1/2 clinical study of SIG-001 in individuals with severe or moderately severe hemophilia.

Interested in working in the hematology field, but don’t think that medical school is right for you? Consider an allied health field, like genetic counseling or physical therapy. Recently, NHF sat down with Mitch Starkman, a physical therapist, and Stefanie Dugan, a genetic counselor, to discuss their career paths and professional experiences. Read these Q&As and watch their respective videos to learn more! 


The onset of childbirth and the postpartum period are times when women with von Willebrand disease (VWD) are at an increased risk for excessive bleeding, exposing them to further, and in some instances, serious complications. While there exist therapies with VWD-specific indications, it is not uncommon for these patients to still experience excessive bleeding while receiving treatment. These scenarios are challenging as there is sparce clinical data and a subsequent lack of clear guidance on the optimal management of bleeding in these particular settings.

uniQure and CSL Behring recently announced that etranacogene dezaparvovec, an investigational hemophilia B gene therapy currently in clinical trials, has achieved a primary endpoint of “non-inferiority” in annualized bleeding rate (ABR) 18-months post administration. The non-inferiority component of a clinical trial measures whether the investigational therapy is not worse/less efficacious than a currently available product. In this case, etranacogene dezaparvovec is being compared to baseline factor IX prophylactic replacement therapy as part of the phase III HOPE-B pivotal trial.




Sigilon Therapeutics today reported a new development in the company’s phase 1/2 clinical study of SIG-001 in individuals with severe or moderately severe hemophilia. This follows the events of this past summer when the U.S. Food and Drug Administration (FDA) placed a clinical hold on the trial due to an unwanted inhibitor response in one of the three patients participating in the study.

Historically, data on infants and toddlers (ITs) with von Willebrand disease (VWD), particularly relevant to bleeding patterns, has been lacking. To address this absence of data, a team of researchers from the U.S. Hemophilia Treatment Center Network (USHTCN) and the Centers for Disease Control and Prevention conducted retrospective analysis of HTC patients with VWD who are less than two years of age. The data were obtained through the USHTCN. The results were published in the journal Blood Advances.

Spark Therapeutics announced updated data from a phase 1/2 clinical trial of SPK-8011

For more than 40 years, the hemophilia treatment center (HTC), the U.S. HTC Network (USHTCN), and its model of integrated, patient-centered care has been essential in helping people with hemophilia (PWH) achieve optimal health outcomes.

button pdf

NHF Statement In Response to New Michigan Law:

Media Contact:

Ilana Ostrin

212 328 3769

The Indiana Hemophilia & Thrombosis Center and Partners in Bleeding Disorders Education Program have announced a new education program designed for physicians who practice at federally funded hemophilia treatment centers (HTCs) in the U.S. The Partners Physician Academy (PPA) will focus specifically on supporting and accelerating third-and-fourth year fellows and junior physicians for up to two years post-fellowship.

NHF is very pleased to announce that the Kevin Child Scholarship (KCS) application deadline has been set for August 2, 2021! The scholarship is named in tribute to Kevin Child, who died of AIDS in 1989, shortly before graduating from college. It is awarded annually to a student with hemophilia who is about to enter or currently enrolled in a program of higher education.  

Shared decision making (SDM), the concept by which patients and healthcare providers work closely together to arrive at personalized treatment-related decisions, can have significant positive impacts for individuals with hemophilia.

A new paper published in The Journal of Haemophilia Practice advances the concept of shared decision making (SDM) between the hemophilia patient and their healthcare provider (HCP).

A new article, “Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia,” was published in the Journal of Clinical Medicine (JCM).

In this review, the authors discuss some of the primary considerations relevant to investigational gene therapies that employ adeno-associated viral (AAV) vectors, with particular focus on immunogenicity and genotoxicity – the former denotes the ability of a foreign substance to trigger an immune response, while the latter refers to a substances ability to damage genetic material.

8031 West Center Road
Suite 304
Omaha, NE 68124

© National Hemophilia Foundation - Nebraska Chapter 2022

Crafted by Firespring