News
Study Suggests Racial and Ethnic Disparities in Hemophilia and ITI
Industry News & Research, Living with a Bleeding Disorder
Findings of a new study uncovered specific disparities in hemophilia care by focusing on the use of immune tolerance therapy (ITI) in…
First Patient Dosed with Hemophilia Gene Therapy Candidate ASC618
Gene Therapy, Industry News & Research
The first patient has been dosed in ASC Therapeutics clinical trial of ASC618, the company’s investigational AAV8 vector-based gene therapy for patients…
The Liver Disease That’s on the Rise
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
Here’s why people with bleeding disorders need to know about it. Have you heard of metabolic dysfunction-associated steatotic liver disease, or MASLD?…
Study Results Encourage Discussion of Therapeutic Options for VWD Patients with Menorrhagia
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder, Women with Bleeding Disorders
A new study investigated therapeutic options for von Willebrand Disease patients to help mitigate the negative health impacts of heavy menstrual bleeding. …
Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
NBDF’s Medical and Scientific Advisory Council issued two new documents making recommendations on criteria for hepatocellular cancer (HCC) screening and helping hemophilia…
New Hemophilia Gene Therapy Video Presentations Available
Industry News & Research
The National Bleeding Disorders Foundation (NBDF) is pleased to announce the availability of a series of video presentations on fundamental topics relevant…
Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Two new documents have been issued by the NBDF’s Medical and Scientific Advisory Council (MASAC), which periodically writes recommendations and advisories on…
Hepatocellular Cancer Screening and Gene Therapy Preparedness the Focus of New Recommendations
Gene Therapy, Industry News & Research
Two new documents have been issued by the NBDF’s Medical and Scientific Advisory Council (MASAC), which periodically writes recommendations and advisories on…
Study Results Encourage Discussion of Therapeutic Options for VWD Patients with Menorrhagia
Industry News & Research
While the majority of women with von Willebrand disease (VWD) will experience heavy menstrual bleeding (HMB) often associated negative impacts on overall…
New publications provide guidance to increase identification of PWBDs
Industry News & Research
One of the World Federation of Hemophilia’s (WFH) 2021-2025 strategic priorities is to enhance the identification and diagnosis of people with bleeding…
Paper Highlights Treatment for Ultra-Rare Type 1 Plasminogen Deficiency
Industry News & Research
Type 1 plasminogen deficiency, also known as hypoplasminogenemia, is a very rare genetic disorder. A recent publication in the journal Haemophilia, represents a…
New Study Looks at Hemophilia Survival Rate Disparities
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
Black men experience much lower hemophilia survival rates compared to their white counterparts. Results of a new study published in the journal Haemophilia are…
Investigational T-Cell Therapy for Hemophilia A Inhibitors Receives Orphan Drug Designation
Industry News & Research
Orphan drug designation is awarded to therapies designed to treat rare diseases affecting fewer than 200,000 people in the United States. The…
International Hemophilia Survey Suggests Psychosocial Challenges and Lack of Shared Decision Making
Health and Well Being, Industry News & Research
Some compelling results from the ‘Hemophilia Life Stages and Changes Global Survey’ were recently announced in a new press release. The survey…
Pfizer Announces Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial
Industry News & Research
Marstacimab (PF-06741086) is under development for the treatment of hemophilia A and hemophilia B. It’s administered through intravenous or subcutaneous route. It…
Bypassing Agents for Hemophilia Explained
Industry News & Research, Living with a Bleeding Disorder
Learn about bypassing agents as treatment for inhibitors and whether they might be right for you. One of the most problematic issues…
Investigational T-Cell Therapy for Hemophilia A Inhibitors Receives Orphan Drug Designation
Industry News & Research
Orphan drug designation is awarded to therapies designed to treat rare diseases affecting fewer than 200,000 people in the United States. The…
Gene Therapy in Hemophilia: Come Find Out What All the Buzz is About
GLHF News, Industry News & Research
From Curiosity To Eligibility And Beyond! Gene therapy has arrived! Join us for a one-hour presentation to hear how gene therapy is…
Depression, anxiety prevalent among hemophilia patients: Study
Health and Well Being, Industry News & Research, Living with a Bleeding Disorder, Men with Bleeding Disorders
Analysis focused on 90 men with hemophilia A and B in Turkey by Lindsey Shapiro, PhD | September 15, 2023 About a quarter (24.4%) of…
Paper Highlights Treatment for Ultra-Rare Type 1 Plasminogen Deficiency
Industry News & Research
Type 1 plasminogen deficiency, also known as hypoplasminogenemia, is a very rare genetic disorder. A recent publication in the journal Haemophilia, represents a…
Learn More About Patient Assistance Programs
Advocacy & Legislation, Health and Well Being, Industry News & Research, Living with a Bleeding Disorder
There are several programs and resources designed to assist individuals and families grappling with the financial burden of living with an inheritable…
Gynecologists Have Significant Part to Play in Shrinking Diagnostic Gaps
Industry News & Research, Living with a Bleeding Disorder, Women with Bleeding Disorders
A new commentary, “Closing the Diagnostic Gap in Adolescents and Young Adult Women with Bleeding Disorders,” published in the August issue of…
Statement: HFA Responds to Latest Gene Therapy Announcement
Gene Therapy, Industry News & Research
Yesterday, the U.S. Food and Drug Administration approved Roctavian (valoctocogene roxaparvovec), the first gene therapy for hemophilia A. This new treatment option, for the treatment of…
UPDATE: Takeda to Voluntarily Replace Certain BAXJECT® II Reconstitution Devices
Industry News & Research
Takeda, in agreement with the U.S. Food and Drug Administration (FDA), has decided to voluntarily replace BAXJECT® II reconstitution devices produced by…