News

NHF Assumes Leadership of the American Plasma Users Coalition

Advocacy & Legislation, Industry News & Research

NHF Assumes Leadership of the American Plasma Users CoalitionThe organization’s public policy representatives offer new perspectives in the mission for blood and…

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New FIX Chromogenic Assay Launched by Precision Biologics

Industry News & Research, Uncategorized

This assay is designed to help determine factor IX activity levels in the plasma of individuals with hemophilia B who are two…

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FDA Accepts BioMarin’s Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Adults with Severe Hemophilia A

Industry News & Research, Uncategorized

If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A PDUFA Target Action Date is March 31, 2023….

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Positive Gene Therapy Results

Industry News & Research, Uncategorized

Pfizer had positive results from the Phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult…

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Common Questions About von Willebrand Disease (VWD)

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

It’s the most prevalent, yet lesser known, bleeding disorder. Here’s what you need to know about VWD. by: Michael Hickey Of the…

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NHF Fellowship: 50 Years of Insights, Innovation, and Breakthroughs

Industry News & Research, Research, Uncategorized

NHF’s Judith Graham Pool Postdoctoral Research Fellowship program turns 50. We spoke with five awardees about how the fellowship shaped their careers….

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Can People with Bleeding Disorders Take Blood Thinners?

Industry News & Research, Living with a Bleeding Disorder, Uncategorized

by: Donna Behen Blood Safety Millions of Americans who have or are at high risk for heart disease regularly take anti-platelet drugs…

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Telehealth for the Bleeding Disorders Community

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

Telemedicine provided vital access to blood and bleeding disorders care during the pandemic. What does the future hold for this technology? Remote…

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Hemophilia Federation of America Outlines Year-End Omnibus Spending Bill with Important Health Provisions Affecting Medicaid, Telehelath, New Drugs and More

Advocacy & Legislation, Industry News & Research

Hemophilia Federation of America’s Word from Washington outlines a massive year-end omnibus spending bill containing numerous important health provisions affecting Medicaid, telehealth,…

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Pfizer and Sangamo Therapeutics Announce Phase 3 Trial of Investigational Gene Therapy for Hemophilia A Has Re-Opened Recruitment September 23, 2022 Share on facebook Share on twitter Share on linkedin

Industry News & Research, Living with a Bleeding Disorder

Pfizer and Sangamo Therapeutics announced that the Phase 3 AFFINE study evaluating giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately…

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HFA Statement on FDA Approval of First-Ever Gene Therapy for Hemophilia B

Industry News & Research, Living with a Bleeding Disorder

Five years ago, Hemophilia Federation of America articulated a broad vision that includes advocating for improved care and quality of life for…

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FDA Approves First Gene Therapy to Treat Adults with Hemophilia B

Industry News & Research, Living with a Bleeding Disorder, Uncategorized

On November 22, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus (AAV) vector-based gene therapy. It is…

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BioMarin Receives Conditional Approval in Europe for Gene Therapy

Industry News & Research

The European Commission granted conditional approval to BioMarin’s Hemophilia A gene therapy Valoctocogene Roxaparvovec which will be known as Roctavian. With this…

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NHF Calls on CMS to Address Copay Accumulator Adjustors in the 2024 Notice of Benefit and Payment Parameters

Advocacy & Legislation, Industry News & Research

In early October, NHF joined members of The All Copays Count Coalition (ACCC), sending a letter signed by 73 leading patient advocacy and…

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Recruitment Reopened for Phase 3 AFFINE Gene Therapy Trial

Industry News & Research

This trial is evaluating the efficacy and safety of a single infusion of an investigational gene therapy in individuals with moderately severe…

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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,…

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Statement from Patient Groups in Response to Court Ruling on Cost-Free Coverage of Preventive Health Services

Industry News & Research, Living with a Bleeding Disorder

HFA and 25 other patient groups issued the following statement in response to Wednesday’s court ruling in Braidwood v. Becerra (a lawsuit formerly known…

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Rare Factor X Deficiency Survey Shows Disparity in Care

Industry News & Research

Initial data from the survey were presented at the recent Thrombosis and Hemostasis Summit of North America and NHF’s Bleeding Disorders Conference…

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FDA Grants Priority Review for Sanofi’s BIVV001

Industry News & Research

BIVV001 is a recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once weekly…

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von Willebrand Disease (VWD) and Iron Deficiecy – Speaker: Bethany Samuelson, M.D.

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

In this video, Dr. Bethany Samuelson Bannow, of Oregon Health & Science University, discusses the role of iron in the body, the…

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Gene Therapy and Bleeding Disorders: Making Sense of It All

Health and Well Being, Industry News & Research

Gene Therapy is definitely a dominant topic in the bleeding disorders community, with new articles being published regularly. Read some of the…

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Supreme Court Decision and Genetic Testing

Health and Well Being, Industry News & Research

by: Laurie Kelley Thoughts on how the Supreme Court decision on abortion might affect genetic testing outcomes. Read more here. The recent…

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Does Gene Therapy Face Uphill Battle? Gene Therapies for Hemophilia Treatment Will Face Strong Market Headwinds, Research Suggests

Industry News & Research

Solid market position of Hemlibra and strong prescriber interest in RNAi and TFPI treatment options, combined with patient concerns regarding gene therapies,…

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Gene Therapy and Bleeding Disorders: Where Are We Now, and What’s on the Horizon?

Industry News & Research

The National Hemophilia Foundation’s 16th Workshop on Novel Technologies and Gene Transfer for Hemophilia, held in November, addressed these questions and more….

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