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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
