Stay informed with the latest updates for the inheritable blood disorders community.

Industry
BioMarin Provides Update Including Next Steps for Investigational Gene Therapy
Trial Data for Roche Investigational SubQ Hemophilia Therapy Presented…
Medical
A group of health care providers standing together
New Accredited Activity Explores Individualized Care for von…
Medical
Research Blood Disorder Vial
FDA Approved Test Measures Concizumab-mtci in Hemophilia Patients
Industry
Gene Therapy Helix
Integration of Spark into Roche Group Set, Hemophilia Gene…
Government Relations Update
Government Relations Update – May 2025
Press Releases
NBDF graphic with 8 blood drops
NBDF’s Keri Norris Finalist for Fierce Healthcare Award
NBDF & Community
A group of people discussing mental health
The NFRC Features Mental Health Resources for Bleeding Disorders Community…
NBDF & Community
A researcher holding a beaker and dropper
UPDATE: NBDF’s Venture Philanthropy Fund: Pathway to Cures
Medical
A doctor with a father and son
FDA Grants Jivi Expanded FDA Indication for Younger Hemophilia A Patients…
Medical
A woman in a lab conducting research by examining blood
NEJM Publishes Follow-up Data on Hemophilia B Gene Therapy Product
Peer-Reviewed Journal Summaries
First Patient Dosed in Freeline’s Dose-Confirmation Trial for Hemophilia B Gene Therapy
New Paper Features Hemophilia Gene Editing Lexicon
NBDF & Community
Slowdown of Gene Therapy Research Spurs Call to Action
Advocacy & Legislative
The U.S. Capitol Building
Government Relations Update - April 2025
Peer-Reviewed Journal Summaries
A group of diverse healthcare providers
ASH Led Initiative Focuses on Greater Representation in Hematology…
NBDF & Community
Doctor with a patient, reviewing resources
NFRC Features von Willebrand Disease Resources