News

First Hemophilia B Patient Dosed with Gene Therapy Product

Industry News & Research

HEMGENIX is indicated for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or…

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Pfizer Announces Positive Results from Hemophilia A and B Trial

Industry News & Research

Pfizer Inc. today announced their Phase 3 BASIS clinical trial evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and…

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HFA Launches Clinical Trials Search Site

Industry News & Research

Hemophilia Federation of America has launched a new resource and search feature for clinical trials for patients with bleeding disorders.  “Patients and families have asked for…

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CSL Behring Announces the First Patient has Received FDA ApprovedHEMGENIX® (etranacogene dezaparvovec-drlb) for Hemophilia B

Industry News & Research

HEMGENIX®, the first and only FDA approved gene therapy for hemophilia B,has been proven to elevate and sustain factor IX levels for…

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Announcement from NHF CEO and President

Industry News & Research

Dr. Leanord Valentino announces his impending retirement. His statement reflects on the progress and initiatives made during his three and a half…

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FDA Finalizes Blood Donor Eligibility Guidance

Industry News & Research

On May 11, 2023, the U.S. Food and Drug Administration released final guidance providing recommendations for evaluating blood and plasma donor eligibility using individual risk-based…

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Pfizer Highlights Phase 3 Trial Results of Marstacimab

Industry News & Research

Marstacimab is a laboratory-engineered monoclonal antibody developed to treat hemophilia A and B patients, with or without inhibitors. Pfizer has announced positive…

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Gene Therapy as a Novel Approach to Achieving ITI in FVIII Inhibitors Reviewed

Industry News & Research

The review provides a comprehensive overview of the persistent challenges and current mitigation strategies related to treating and managing hemophilia patients with…

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FDA Finalizes Blood Donor Eligibility Guidance

Industry News & Research

On May 11, 2023, the U.S. Food and Drug Administration released final guidance providing recommendations for evaluating blood and plasma donor eligibility using individual risk-based…

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Pfizer Highlights Phase 3 Trial Results of Marstacimab

Industry News & Research

Marstacimab is a laboratory-engineered monoclonal antibody developed to treat hemophilia A and B patients, with or without inhibitors. Pfizer has announced positive…

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The Low Cost of Being Female with a Bleeding Disorder?

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

Female hemophilia A patients and carriers have more frequent bleeds than males, yet a smaller proportion receive factor to prevent or stop them, a U.S….

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Now Enrolling: Own Your Path

Industry News & Research

Are you a male, 18-29 years old, and on a prophy regimen for hemophilia treatment? You may qualify for a new program!…

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Four New Documents from MASAC

Industry News & Research

The new guidelines cover recommendations for physical therapy, gene therapy readiness, vaccine guidance, and treatment. Download here. Source: National Hemophilia Foundation

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New in Research: Updates on the “My Life Our Future” Project

Industry News & Research

Learn how this project from NHF, the American Thrombosis and Hemostasis Network, Bloodworks Northwest, and Bioverativ/Sanofi helped inform people with hemophilia on…

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Inhibitor Risk Paper Informed by Genotyping Project

Industry News & Research

Between 2013-2017, the “My Life Our Future” (MLOF) project offered eligible individuals with hemophilia free genotyping, which is historically hard to access,…

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Important: Action Needed Regarding Medicaid and Badgercare Eligibility

Advocacy & Legislation, Industry News & Research

As you may have heard, the Medicaid and CHIP (called Badgercare in Wisconsin) programs are re-starting eligibility reviews. Community members may receive…

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FDA Requests Additional Information on Concizumab from Novo Nordisk

Industry News & Research

The company received a Complete Response Letter from the FDA seeking more information relevant to their BLA for concizumab. Novo Nordisk has…

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Updates on Generic Desmopressin Product

Industry News & Research

Desmopressin has the capacity to boost both factor VIII and von Willebrand factor levels. In July 2020 Ferring Pharmaceuticals suspended manufacturing of…

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New Survey Spotlights Ongoing Concerns for People Living with Hemophilia B

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

Carefully controlled schedules. Undercurrents of uncertainty. Persistent pain. People who are living with hemophilia B, and those who love them, face a…

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HTCs Participate in New CDC Community Counts Data Collection for COVID-19

COVID-19, Industry News & Research

Patients with bleeding orders may be asked some new questions during their next comprehensive clinic visit. COVID-19 information will now be collected…

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Sanofi Announces Publication of Two Phase 3 Fitusiran Trials

Industry News & Research

Fitusiran is being investigated for the prophylactic treatment of adults and adolescents with hemophilia A or B. Sanofi recently announced that results…

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Hemochromatosis Classification: Update and Recommendations by the BIOIRON Society

Industry News & Research

This is a related article to: Hemochromatosis redefined Abstract Hemochromatosis (HC) is a genetically heterogeneous disorder in which uncontrolled intestinal iron absorption may…

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FDA approves once-weekly ALTUVIIIO™, a new class of factor VIII therapy for hemophilia A that offers significant bleed protection

Industry News & Research, Living with a Bleeding Disorder

FDA approves once-weekly ALTUVIIIO™, a new class of factor VIII therapy for hemophilia A that offers significant bleed protection Paris and Stockholm – February 23, 2023 – The U.S. Food and Drug Administration (FDA) has…

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Introducing the WFH Gene Therapy Registry

Industry News & Research

The new registry will enhance knowledge of gene therapy and help keep patients safe. The World Federation of Hemophilia (WFH) is a nonprofit organization…

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