Federal  Updates:

Pfizer has announced positive results from their global phase 3 BASIS clinical study of marstacimab, the company’s investigational, subcutaneous therapy that targets an anticoagulant protein known as tissue factor pathway inhibitor (TFPI).

Dear National Hemophilia Foundation Community, Staff and Friends,

When Saylor Behrens' biology professor told her class that hemophilia is the result of incest, she grew angry. She knew better. Her younger brother, Wyatt, has hemophilia A.

It wasn't just the false narrative being spread that made her indignant- it was the sheer lack of awareness about hemophilia that morphed her brother's bleeding disorder into a scary, incredibly rare condition that could be brushed off. 

Every day, chapters across the country work with the blood and bleeding disorders community around them. Since 2021, Jacob Murdock has served as Chapter Executive Director in Nevada. He took time out of his busy schedule to tell us why loves the Nevada Chapter.

How long has your Chapter been serving the community?

We were founded as the Hemophilia Foundation of Nevada in 1990. We became a national chapter of NHF in 2011. The chapter was founded by Renee Paper, a nurse and a pioneer in the treatment of women with bleeding disorders.

The Rare Diseases Diversity Coalition (RDDC) is currently seeking applicants for its 2023 Rare Disease Fellowship Program, which is aimed at inspiring the next generation of the medical workforce to work in the rare disease space and achieve greater health equity for people of color living with rare diseases.

The Fellowship allows rare disease organizations to collaborate with fellows to better understand the health impacts of that disease on communities of color. In addition, the Fellow will gain a greater awareness of the challenges of living with rare diseases

NHF’s Medical and Scientific Advisory Council (MASAC) recently issued four new documents, which are now available for easy access.

In July 2020 Ferring Pharmaceuticals suspended manufacturing of the nasal spray  STIMATE®, a popular product used to treat certain bleeding disorders. The suspension, and subsequent pharmacy level recall, were in response to a packaging seal issue that caused evaporation, a potential hazard to patients who could be exposed to dangerously high concentrations of the product’s active ingredient desmopressin acetate.

Between 2013-2017, the “My Life Our Future” (MLOF) project offered eligible individuals with hemophilia free genotyping, which is historically hard to access, expensive, and not covered by insurance.

Novo Nordisk has notified the National Hemophilia Foundation that they have received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for their investigational, subcutaneous therapy concizumab.

Federal Updates:

FOR IMMEDIATE RELEASE

Ilana Ostrin

Senior Director of Public Relations and Communications

The foundation's staff is comprised of dedicated people use their talents to help the blood and bleeding disorders community. One of them is Karina Lopez, MPH! Karina joined us to talk about her progression from an internship to a full-time position as a Project Management Specialist at the organization and one of the projects she's most proud of during her time here.

Watch the interview or read the full conversation below.

Having everyone's perspectives is critical to forming solid research or a well-rounded program. But how do you get the perspectives of people who are not already be involved, whether by choice or circumstance? HEDI Program Specialist, Marissa Melton, starts a conversation on how to engage unengaged communities.

As NHF continues to celebrate it's 75th Anniversary, the organization sat down with community member Eldon Ham to learn more about his story.

For Immediate Release

Renee Peck

Pathway to Cures
rpeck@pathwaytocures.org

212-328-3790

New York, NY, February 27, 2023 – Pathway to Cures (P2C), a venture philanthropy fund focused on transformative treatments for inheritable blood disorders, announces the appointment of its investment committee.

Coalition Advocates for Substance Use Disorder/Behavioral Health Services for Patients With Bleeding Disorders

Sanofi recently announced that results of two clinical studies of their investigational therapy fitusiran were recently published in The Lancet and The Lancet Haematology journals. Both phase 3 trials were evaluating the efficacy and safety of fitusiran for the prophylactic treatment of adults and adolescents with hemophilia A or B. The enrolled participants, all of whom are 12 years of age and up, had been previously treated with existing factor replacement therapy or bypassing agent (BPA) prophylaxis.

Federal Updates:

Washington Days Recap: Nearly 400 volunteer advocates attended Washington Days March 8-10th and participated in 235 meetings with Members of Congress and staff.  The focus of this year was soliciting cosponsors for H.R. 830, the HELP Copays Act, and securing level funding for the federal hemophilia programs.

During legislative session, NHF's chapter network, public policy staff, and the bleeding disorders community at large has been hard at work advocating across the United States! For the month of March, here are some advocacy and policy highlights from the beginning of this year:

To end Bleeding Disorders Awareness Month and celebrate National Doctors Day, Dr. Aaron Chen shares his story of how living with thalassemia inspired his calling as a physician. Watch his interview or read the full transcript below.

April is National Minority Health Month. To learn about the experiences of the bleeding disorders community in an island state, Chloe, a member of the communications team, sat down with Kyra Calbero, Interim Executive Director of the foundation's Hawaii chapter.

Watch a video below that summarizes the conversation, or read the whole transcript.

The foundation's staff is comprised of dedicated people use their talents to help the blood and bleeding disorders community.

A new member of the team is Chloe Fellwock, a communications assistant who joined began her tenure in June 2022. A member of the communications team sat down with her to talk about working this role as her first job out of college, what she's learned, and her favorite moments with the team.

Akron Pharmaceuticals, one of several companies that manufactures and markets the product aminocaproic acid, recently filed for chapter 7 bankruptcy and subsequently closed.