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uniQure Announces Findings from HCC Case Investigation in HOPE-B Trial for Gene Therapy

Industry News & Research

comprehensive investigation into a case of hepatocellular carcinoma (HCC) diagnosed in one patient participating in the phase III HOPE-B pivotal trial of etranacogene dezaparvovec, the company’s investigational gene therapy candidate…

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NHF Statement on HELP Copays Act

Advocacy & Legislation, Industry News & Research

…last night (11/1/2021). This new legislation aims to support the more than 133 million Americans (40%) living with a chronic health condition, by ensuring that all payments—whether they come directly…

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New Data Analysis Sheds Light on Bleeding Patterns in Young VWD Patients

Health and Well Being, Industry News & Research, Living with a Bleeding Disorder

…characteristics, bleeding episodes, and complications. A review of birth and delivery data showed that 86% were delivered at full term, 82% were of normal weight, and 89% were considered to…

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Takeda Announces Approval of Prophylactic Indication for VWD Therapy

Industry News & Research

…therapy on the market. “VWD is a complex disease where both patients and providers may experience stress and uncertainty due to the unpredictable disease course and limited treatment options,” said…

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New Government Funded Research Program Created to Help Improve Gene Therapies for People with Hemophilia A

Industry News & Research

A group of investigators are embarking on a new research program designed to unpack some of the outstanding fundamental questions associated with current approaches to gene therapy for hemophilia A….

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NHLBI Grant Supports Scientific Research to Enhance Hemophilia A Gene Therapies

Industry News & Research

The new grant will fund basic and translational studies to improve gene therapies for hemophilia A. A group of investigators are embarking on a new research program designed to unpack…

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Record-Keeping Tips for the Bleeding Disorders Community

Health and Well Being, Living with a Bleeding Disorder

…you need to demonstrate clearly how your bleeding disorder affects you. A record of all your factor infusions, bleeding episodes, and lab tests will show just that, helping you build…

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BioMarin Shares Community Update for Valoctocogene Roxaparvovec

Industry News & Research

Biomarin recently provided an “Update for the Hemophilia Community,” on the ongoing clinical trial program for its investigational hemophilia A gene therapy, valoctocogene roxaparvovec. It comprises a brief overview of…

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Additional Health Risks for Men with Hemophilia

Living with a Bleeding Disorder, Men with Bleeding Disorders

…the risk of high blood pressure (hypertension) among men with hemophilia, a U.S. study found, published in the journal Haemophilia. Visit here for more info. Source: LA Kelley Communications, Inc….

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FDA Approves First Gene Therapy to Treat Adults with Hemophilia B

Gene Therapy, Industry News & Research, Living with a Bleeding Disorder

for routine FIX replacement prophylaxis, and a 54% reduction in ABR compared to baseline. The most common adverse reactions associated with Hemgenix treatment were liver enzyme elevations, headache, mild infusion-related…

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Men with Bleeding Disorders

Men can and do have bleeding disorders. Despite long-standing misconceptions about bleeding disorders in men, men’s voices and experiences are now being taken more seriously. Study Finds Men with Hemophilia…

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WFH Gene Therapy Registry Goes Live

Industry News & Research, Living with a Bleeding Disorder

…designed to collect critical data on patients who have received a gene therapy through a clinical trial or via a product that has already come to market. By employing such…

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BioMarin Announces FDAโ€™s Extended Review of Investigational Hemophilia Gene Therapy

Industry News & Research

…data and therefore set a new PDUFA* Target Action Date of June 30, 2023 – the company had previously communicated that this data submission could be qualified as a Major Amendment.  “We are…

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Pfizer Highlights Phase 3 Trial Results of Marstacimab

Industry News & Research

study of marstacimab, the company’s investigational, subcutaneous therapy that targets an anticoagulant protein known as tissue factor pathway inhibitor (TFPI). Marstacimab is a laboratory-engineered monoclonal antibody developed to treat hemophilia…

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FDA Finalizes Blood Donor Eligibility Guidance

Industry News & Research

…end user. In advance comments on the revised guidance, APLUS commended FDA’s transition to an individual risk-based assessment; advocated for ongoing surveillance and data collection activities around donor eligibility; and urged continued…

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Pfizer Highlights Phase 3 Trial Results of Marstacimab

Industry News & Research

study of marstacimab, the company’s investigational, subcutaneous therapy that targets an anticoagulant protein known as tissue factor pathway inhibitor (TFPI). Marstacimab is a laboratory-engineered monoclonal antibody developed to treat hemophilia…

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FDA Finalizes Blood Donor Eligibility Guidance

Industry News & Research

…end user. In advance comments on the revised guidance, APLUS commended FDA’s transition to an individual risk-based assessment; advocated for ongoing surveillance and data collection activities around donor eligibility; and urged continued…

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Inequities in Thrombosis and Hemostasis Lab Testing the Focus of Illustrated Review

Industry News & Research

…reduce health disparities; foster and fund inclusive health research via recruitment of diverse participants to promote generalizability of research findings; prioritize research areas of extreme health disparity, particularly at vulnerable…

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Bleeding Disorders: What Your Pain Is Trying to Tell You

Health and Well Being, Living with a Bleeding Disorder, Pain Management

…Listen The science of pain is complex, and individuals experience it differently, but the core concept is straightforward. The International Association for the Study of Pain (IASP) defines it as “an unpleasant sensory and

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First Hemophilia A Gene Therapy Approved

Industry News & Research

…year. Most patients were given corticosteroids to suppress the immune system so that the gene therapy would be effective. For more info read here. Source: LA Kelley Communications, Inc., Communique’…

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Better Bleeding Disorders Care Begins with You

Health and Well Being, Living with a Bleeding Disorder

and patient community can come together to create a national blueprint of patient-centered research with quantifiable outcomes.” Spearheading what the future of research should look like was a logical and

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Investigational T-Cell Therapy for Hemophilia A Inhibitors Receives Orphan Drug Designation

Industry News & Research

…release, the phase 1/2a study will investigate the safety and efficacy of TI-168 in patients with congenital hemophilia A and an inhibitor. Participants will also be treated prophylactically with emicizumab…

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Pfizer Announces Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial

Industry News & Research

…pathway inhibitor (TFPI). The study demonstrates statistically significant and clinically relevant reduction in annualized bleeding rate compared to prophylaxis and on-demand intravenous regimens. Marstacimab, if approved, has the potential to…

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Investigational T-Cell Therapy for Hemophilia A Inhibitors Receives Orphan Drug Designation

Industry News & Research

…release, the phase 1/2a study will investigate the safety and efficacy of TI-168 in patients with congenital hemophilia A and an inhibitor. Participants will also be treated prophylactically with emicizumab…

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