PO Box 4214
Morgantown, West Virginia 26504
Phone 304-212-2616

If you want to be in the know about what’s going on at our organization, you’ve come to the right place!

  • Keep up to date with the chapter and look back on previous events.

  • Find resources and hear stories from community members in this newsletter just for for women, girls, and people with the potential to menstruate.

Be sure to check back regularly to get our latest news 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,

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, plus 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.

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.

Answering Your Questions on the Proposed Change to the FDA's Blood Donation Policy

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

Researchers from the Children’s Healthcare of Atlanta and Emory University recently published the results of a small study investigating the subject of guilt in mothers of children with hemophilia (CWH).

March 15, 2023 – The National Hemophilia Foundation (NHF) today announced that a series of manuscripts central to advancing patient-focused research for the inherited bleeding disorders (IBD) community have been published in the journal Expert Review of Hematology. These papers describe initial recommendations from six multi-disciplinary working groups established to evaluate and address the most urgent priorities for the community, and will form the basis for NHF’s community-driven National Research Blueprint (NRB).

Spring is fast approaching, which means it’s time to talk scholarships!

This Bleeding Disorders Awareness Month, hematologists Ronak Mistry, DO and Dan Hausrath, MD start a conversation around their experiences being early career physicians, and launching a podcast. Watch their video and read a transcript.

Learn more about Bleeding Disorders Awareness Month here.

Biomedical and pharmaceutical companies who intend to bring a product to market must demonstrate safety and efficacy of those products via closely monitored preclinical and clinical studies.

On March 6th BioMarin announced that it received a notice from the U.S. Food and Drug Administration (FDA) indicating that the agency has extended their review of the company’s Biologics License Application for ROCTAVIAN™ (valoctocogene roxaparvovec).

[NOTE: As part of NHF’s Bleeding Disorders Awareness Campaign, NHF CEO Dr. Len Valentino has invited community members and medical professionals to “start the conversation” on gene therapy. The opinions shared in these pieces do not necessarily reflect the views of the foundation.]

By Ray Stanhope

[NOTE: As part of NHF’s Bleeding Disorders Awareness Campaign, NHF CEO Dr. Len Valentino has invited community members and medical professionals to “start the conversation” on gene therapy. The opinions shared in these pieces do not necessarily reflect the views of the foundation.]

[NOTE: As part of NHF’s Bleeding Disorders Awareness Campaign, NHF CEO Dr. Len Valentino has invited community members and medical professionals to “start the conversation” on gene therapy. The opinions shared in these pieces do not necessarily reflect the views of the foundation.]

NHF is pleased to announce that its collaboration with Medscape continues with a new accredited educational activity designed to help healthcare providers fully incorporate gene therapy into the collaborative care model.

 

National Hemophilia Foundation Launches “Pathway to Cures,” a Venture Philanthropy Fund to Accelerate the Development of Transformational Therapies and Technologies for Inheritable Blood Disorders

FOR IMMEDIATE RELEASE

MEDIA CONTACT:
iostrin@hemophilia.org

 

uniQure recently announced publication of the clinical trial results associated with approval of the hemophilia gene therapy product

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 February, here are some advocacy and policy highlights from the beginning of this year:

The U.S. Food and Drug Administration (FDA) has approved ALTUVIIIO ™, formerly known as efanesoctocog alfa, for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A.

Washington, D.C. - The APLUS Coalition, an alliance of patient advocacy organizations, has announced a new joint statement in a show of solidarity for those living with conditions requiring the use of blood and/or plasma products. The statement comes after the U.S. Food and Drug Administration (FDA) announced new draft guidance that would implement an individualized risk assessment for all potential donors regardless of their sexual orientation or the gender of their partners. This comes after many years of attention to the deferral criteria for men who have sex with men (MSM).

While it is well understood that individuals with von Willebrand disease (VWD) will experience a variety bleeding episodes throughout their lifetime, the psychosocial impact of these symptoms has received relatively little inquiry. Greater knowledge of these impacts could help inform and support potential mental health screening efforts for VWD patients at U.S. hemophilia treatment centers (HTCs).


To help fill this gap a group of researchers from several HTCs across the country sought to estimate rates of symptomatic depression and anxiety among individuals with VWD.

This year, NHF celebrates its 75th anniversary. And in honor of this incredible milestone, NHF is welcoming a new era for the inheritable blood and bleeding disorders community by creating a more equitable future for all. This new moment in time will include ongoing and reemphasized priorities, such as eliminating barriers to care, fostering opportunities to interact with the community, and increasing diversity and inclusion in research and advocacy efforts.

[This content was shared for public use by the Centers for Medicare & Medicaid Services]

The World Federation of Hemophilia (WFH) has launched a new registry to help monitor the long-term safety and efficacy of hemophilia gene therapies in people with hemophilia across the globe.

Sanofi recently announced newly published clinical trial data for efanesoctocog alfa, the company’s investigational recombinant factor VIII therapy (rFVIII) for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic intravenous infusions.

FOR IMMEDIATE RELEASE

Contact: Ilana Ostrin

212-328-3769

FOR IMMEDIATE RELEASE

Contact:

Ilana Ostrin

212-328-3769

Precision BioLogic Inc., a company specializing in hemostasis diagnostics, recently announced that they have received the requisite clearance from the U.S. Food and Drug Administration (FDA) to launch and market their CRYOcheck™ Chromogenic Factor IX assay.

Precision Biologic Inc., a company specializing in hemostasis diagnostics, recently announced that they have received the requisite clearance from the U.S. Food and Drug Administration (FDA) to launch and market their CRYOcheck™ Chromogenic Factor IX assay.

BioMarin recently shared a community update on their Hemophilia A Clinical Development Program for valoctocogene roxaparvovec, the company’s investigational gene therapy currently under review by the U.S. Food and Drug Administration (FDA). It included a brief clinical trial overview with the following updates:

Takeda recently announced favorable results from a phase 3 study of TAK-755, the company’s investigational enzyme replacement therapy for an ultra-rare blood disorder known as congenital thrombotic thrombocytopenic purpura (cTTP).

FOR IMMEDIATE RELEASE

Ilana Ostrin

Senior Director of Public Relations and Communications
iostrin@hemophilia.org

212-328-3769

 
NHF Announces 2023 Winter/Spring Wednesday Webinar Schedule

The free series will continue in the new year with topics including gene editing experiments, innovative insurance cards, substance use, and more.


 

PO Box 4214
Morgantown, West Virginia 26504
Phone 304-212-2616

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