If you want to be in the know about what’s happening in the bleeding disorders community, you’ve come to the right place!
Be sure to check back regularly to get our latest news updates.
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.]
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
FOR IMMEDIATE RELEASE
MEDIA CONTACT:
iostrin@hemophilia.org
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.
Pfizer recently announced positive top-line results from the phase 3 BENEGENE-2 clinical study, which is currently evaluating the investigational gene therapy fidanacogene elaparvovec for the treatment of adult males with moderately severe to severe hemophilia B.
FOR IMMEDIATE RELEASE
Ilana Ostrin
Senior Director of Public Relations and Communications
iostrin@hemophilia.org
212-328-3769
NHF Assumes Leadership of the American Plasma Users Coalition
The organization’s public policy representatives offer new perspectives in the mission for blood and product safety.
Recruitment is now open for Own Your Path- a program for young adult males with hemophilia who are currently in the transition years between 18-29. We know that maintaining adherence to a prophy regimen can have short and long -term health benefits. This program focuses on helping participants be successful by focusing on education, health coaching, skill building, and relationship building, all within an app-based environment that includes participation incentives.
Additional program benefits include:
Organization set to look back while moving ahead during historic year
Spark has announced updated multi-year results from its phase 1/2 clinical trial of SPK-8011, the company’s investigational gene therapy for hemophilia A. The results were presented at the recently concluded 64th American Society of Hematology (ASH) annual meeting in New Orleans, Louisiana.
One patient changed Dr. Amy Dunn’s entire career the first-year of residency. It was then that Dr. Dunn diagnosed her first patient with hemophilia.
FOR IMMEDIATE RELEASE
December 13, 2022
MEDIA CONTACT:
Ilana Ostrin
212-328-3769
iostrin@hemophilia.org
Organization set to look back while moving ahead during historic year
On November 22, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus (AAV) vector-based gene therapy. It is approved for the treatment of adults with hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Historically, many residential substance use disorder (SUD) facilities have denied admittance to individuals with bleeding disorders – the basis of these denials are most often the use of self-administered, intravenous medications such a as factor replacement therapies. The lack of access to residential addiction treatment facilities, can have very serious, even fatal outcomes for bleeding disorder (BD) patients in acute need of help with their addiction.
MEDIA CONTACT:
Ilana Ostrin
212-328-3769
iostrin@hemophilia.org
FOR IMMEDIATE RELEASE
Currently, there are over 167.5 million women (including individuals who identify as women or have the propensity to menstruate) living in the U.S. An estimated 3% of them are living with a diagnosed inheritable blood or bleeding disorder – and certainly many more are living their daily lives with pain and bleeding but are undiagnosed. Recent changes in access to women and girls’ health care are likely to have detrimental effects for the blood and bleeding disorders community.
Back Again! The Annual Red Tie Soiree Returns to Honor Community Members in San Francisco
On October 15, the Red Tie Soiree, a yearly gala hosted by the National Hemophilia Foundation, was held in-person for the first time in three years. The community event was held for the first-time on the West Coast, welcoming guests to the historic Julia Morgan Ballroom in San Francisco.
Tell us a little bit about yourself!
My name is Dr. Lacramioara Ivanciu; I’m a resident assistant professor of pediatrics in the Perelman School of Medicine at the University of Pennsylvania. In 2010, when I was a postdoctoral fellow in Dr. Rodney Camire’s laboratory, I was awarded NHF’s Judith Graham Pool Postdoctoral Research Fellowship.
Wonderful! What did your research focus on?
Tell us a little bit about yourself!
I’m Dr. Jyoti Mathur. From 2007 to 2009, I was a fellow with the Judith Graham Pool Postdoctoral Research Fellowship. When I was selected for the fellowship, I was a postdoctoral fellow at Stanford Medical School.
What did your research focus on?
Tell us a little bit about yourself!
Hi! My name is Dr. Laura Haynes, and I work at the Life Sciences Institute at the University of Michigan as a research investigator. I learned about the JGP Fellowship through mentors and colleagues and in 2018, I was granted fellowship.
What did your research focus on?
My work focused on the protein engineering of Plasminogen Activator 1 to develop novel regulators of the fibrinolytic and hemostatic pathways.
Tell us a little bit about yourself!
My name is Dr. Semma Patel. I’m an assistant professor at Emory University. I became a JGP Fellow in 2019, when I was working at Emory as an associate academic research scientist. I had learned about the program from my mentor.
What was the focus of your research?
My project investigated the epitopes recognized in the early immune response to Factor VIII.
How did the JGP Fellowship support your research at the time?
Tell us a little bit about yourself!
I’m Dr. Lisa Smith Webb and I am an associate professor of molecular biology and chemistry at Christopher Newport University. And along with being a Better You Know advocate and member of the bleeding disorder community, I’m a proud past JGP fellow! I was a fellow from 2002 to 2004.
How did you hear about the JGP Fellowship? Had you heard of Judith Graham Pool prior to the fellowship?
Tell us a little bit about yourself!
I’m Dr. Satish Nandakumar; I am an assistant professor at the Albert Einstein College of Medicine. I was awarded the Judith Graham Pool Postdoctoral Research Fellowship when I was working as a postdoctoral fellow in 2017. Before that, I completed my graduate work at St. Jude's Children's Research Hospital in Memphis, Tennessee.
How did you hear about the JGP Fellowship?
My mentor told me about the fellowship and the opportunities it could provide my work.
Tell us a little bit about yourself!
I’m Dr. Sean Quinn; I became a JGP fellow back in 2021. Currently, I’m a postdoctoral research fellow at the Children’s Hospital of Philadelphia. I heard about the JGP Fellowship from my faculty mentor and other researchers at the Children's Hospital of Philadelphia who applied for the fellowship.
What does your research focus on?
My research examines antibody-mediated FV/FVa resistance as a therapeutic approach for hemophilia.
