Hemophilia B Gene Therapy Trial Data Published
Hemophilia B Gene Therapy Trial Data Published
Hemgenix is currently the only hemophilia B gene therapy approved by the FDA.
Learn More About 2024 Bleeding Disorders Scholarships
Learn More About 2024 Bleeding Disorders Scholarships
The HANDI Resources Center Team is pleased to announce new 2024 scholarship opportunities available for the bleeding disorders community!
Takeda Announces Interim Trial Results for Investigational ITP Therapy
Takeda Announces Interim Trial Results for Investigational ITP Therapy
Primary immune thrombocytopenia is a rare disease autoimmune disorder that causes a significant reduction in platelets and bleeding
Video Celebrates Nurses in Bleeding Disorders Care
Video Celebrates Nurses in Bleeding Disorders Care
The video features interviews with nurses who for decades have cared for patients at hemophilia treatment centers across the United States.
New Hemophilia Fact Sheet Now Available
New Hemophilia Fact Sheet Now Available
The fact sheet includes basic information about hemophilia.
Patient Receives First Hemophilia A Gene Therapy Outside a Clinical Trial
Patient Receives First Hemophilia A Gene Therapy Outside a Clinical Trial
Roctavian™ was approved in the summer of 2023 for the treatment of adults with severe hemophilia A
FDA Approves Pair of Cell-Based Gene Therapies for Sickle Cell Disease
FDA Approves Pair of Cell-Based Gene Therapies for Sickle Cell Disease
Sickle cell disease is a rare condition that includes a group of inherited blood disorders associated with an abnormality in the hemoglobin protein.
FDA Approves Recombinant Therapy for Congenital Thrombotic Thrombocytopenic Purpura
FDA Approves Recombinant Therapy for Congenital Thrombotic Thrombocytopenic Purpura
The U.S. Food and Drug Administration (FDA) has approved a recombinant enzyme replacement therapy for people with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood disorder.
New Hemophilia Gene Therapy Video Presentations Available
New Hemophilia Gene Therapy Video Presentations Available
These talks were originally delivered live and in-person as part of NBDF’s 75th Bleeding Disorders Conference.
Paper Highlights Treatment for Ultra-Rare Type 1 Plasminogen Deficiency
Paper Highlights Treatment for Ultra-Rare Type 1 Plasminogen Deficiency
Type 1 plasminogen deficiency, also known as hypoplasminogenemia, is a very rare genetic disorder
HANDI Team Reminder – Learn More About Patient Assistance Programs
HANDI Team Reminder – Learn More About Patient Assistance Programs
These programs provide assistance for a variety of needs of individuals and families affected by inheritable bleeding disorders.
Gynecologists Have Significant Part to Play in Shrinking Diagnostic Gaps
Gynecologists Have Significant Part to Play in Shrinking Diagnostic Gaps
People with inheritable bleeding disorders who have the potential or ability to menstruate will likely experience especially heavy menstrual bleeding in their lifetime.
Hemophilia A Gene Therapy Product Receives U.S. FDA Approval
Hemophilia A Gene Therapy Product Receives U.S. FDA Approval
Discover ROCTAVIAN™, an FDA-approved gene therapy for hemophilia A. Explore its efficacy and potential to transform treatment.
First Hemophilia B Patient Dosed with Gene Therapy Product
First Hemophilia B Patient Dosed with Gene Therapy Product
CSL Behring announces the first HEMGENIX gene therapy dosing for hemophilia B. Learn more about this milestone and its indications.
Aminocaproic Acid Manufacturer Shuttered Though Not Expected to Trigger Shortage
Aminocaproic Acid Manufacturer Shuttered Though Not Expected to Trigger Shortage
Akorn Pharmaceuticals' bankruptcy won't cause a shortage. Discover its role in treating bleeding disorders and its ongoing production status.
Summit Emphasizes Pharmacovigilance in the Inherited Bleeding Disorders Community
Summit Emphasizes Pharmacovigilance in the Inherited Bleeding Disorders Community
Learn how stakeholders are improving safety communication in the inherited bleeding disorders community. Read more.
FDA Approves ALTUVIIIO™ for Hemophilia A Patients
FDA Approves ALTUVIIIO™ for Hemophilia A Patients
Learn about the FDA approval of ALTUVIIIO™ for hemophilia A patients, offering sustained bleed protection and a single weekly dose.
Sanofi Announces New Efanesoctocog alfa Data Published in the NEJM
Sanofi Announces New Efanesoctocog alfa Data Published in the NEJM
Sanofi's efanesoctocog alfa, a potential hemophilia A treatment, shows promise in NEJM-published clinical trial data. Learn more about this innovative therapy.
Takeda Announces Trial Results on Investigational Therapy for Ultra Rare Blood Disorder
Takeda Announces Trial Results on Investigational Therapy for Ultra Rare Blood Disorder
Learn about the positive results from Takeda's phase 3 trial of TAK-755, an investigational therapy for congenital thrombotic thrombocytopenic purpura.
NHF Announces 2023 Winter/Spring Wednesday Webinar Schedule
NHF Announces 2023 Winter/Spring Wednesday Webinar Schedule
Explore a diverse range of topics in NHF's free Wednesday Webinar series starting January 11, 2023. Learn from experts about gene editing, insurance, and more.
MASAC For You
Accessible consumer-friendly publications from MASAC Guidelines, including emergency department management for hemophilia and bleeding disorders (EN & ES).
Meet Former JGP Fellow, Dr. Hongxia Fu!
Meet Former JGP Fellow, Dr. Hongxia Fu!
Dr. Fu was a JGP research fellow from 2014 to 2016. She is a research fellow in the lab of Dr. Timothy Springer at Boston Children's Hospital/Harvard University. She is also an assistant professor and research group leader at the University of Washington.
Virtual Grand Round Series to Focus on Hemophilia Gene Therapy
Virtual Grand Round Series to Focus on Hemophilia Gene Therapy
This program is presented through a collaboration between the NHF and Medscape.
FDA Grants Priority Review for Sanofi’s BIVV001
FDA Grants Priority Review for Sanofi’s BIVV001
BIVV001 is a recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic intravenous infusions.
PPTA Posts Statement on Monkeypox and Plasma Protein Therapies
PPTA Posts Statement on Monkeypox and Plasma Protein Therapies
PPTA represents more than 1,000 human plasma collection centers in North America and Europe, as well as, the manufacturers of plasma protein therapies.
Prophylactic Therapy for Glanzmann Thrombasthenia Moves Towards Clinical Trials
Prophylactic Therapy for Glanzmann Thrombasthenia Moves Towards Clinical Trials
Glanzmann Thrombasthenia is an ultra-rare inherited bleeding disorder that is characterized by poorly functioning platelets.
HANDI Launches New Microsite!
HANDI Launches New Microsite!
The site includes a series of frequently asked questions posed by HANDI patrons.
Communicating with Providers
YOU are your most important advocate in the health care system. If you have a child, you are also their most important advocate to make your concerns heard and understood.
Clinical Study and New Website to Focus on von Willebrand Disease and Pregnancy
Clinical Study and New Website to Focus on von Willebrand Disease and Pregnancy
This new study was developed to increase understanding of how best to manage bleeding during delivery and the postpartum period in women with VWD.
Sigilon Reports Trial Updates for Investigational Hemophilia A Therapy
Sigilon Reports Trial Updates for Investigational Hemophilia A Therapy
The FDA placed a clinical hold on the company's investigational therapy in July of 2021.
Spark Announces Multiyear Data Update for Investigational Gene Therapy
Spark Announces Multiyear Data Update for Investigational Gene Therapy
This data was drawn from a phase 1/2 clinical trial of SPK-8011, Spark's investigational gene therapy for hemophilia A.
New Paper Highlights Integrated Care Model and the National HTC Network
New Paper Highlights Integrated Care Model and the National HTC Network
This paper provides a comprehensive description of the critical services and dynamic coordinating capacity of HTCs within a regional and national framework.
NHF Awarded NIH Grant for State of the Science Research Summit
NHF Awarded NIH Grant for State of the Science Research Summit
Additional Funding Will Aid Community Collaboration at First-Time Event
Inhibitors
Learn about inhibitors, a complication in bleeding disorders where the immune system reacts to clotting factor treatments. Explore diagnosis and management.