News

The Complexity of Unwinding Medicaid

Industry News & Research

The long-heralded Medicaid “unwinding” has begun. As previously reported, state Medicaid programs have, since the start of the COVID-19 pandemic, operated under a law that…

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BioMarin Update: FDA Extends PDUFA Target Action Date to June 30, 2023

Industry News & Research

BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that it received notice this afternoon from the…

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The Importance of Copay Assistance Protections: Awareness Grows

Industry News & Research, Living with a Bleeding Disorder

For over three decades, March has been designated “Bleeding Disorders Awareness Month.” HFA and member organizations across the country mark the occasion…

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FAQ: Proposed Changes to the FDA Blood Donation Policy

Industry News & Research

Answering Your Questions on the recent FDA blood donation policy revisions. Answering Your Questions on the Proposed Change to the FDA’s Blood…

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Aminocaproic Acid Manufacturer Shuttered Though Not Expected to Trigger Shortage

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

Aminocaproic acid, which helps prevent the premature breakdown of blood clots, is often used to treat more minor bleeds in the nose…

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Summit Emphasizes Pharmacovigilance in the Inherited Bleeding Disorders Community

Industry News & Research

The NHF/HFA Safety Summit yielded a series of recommendations related to product safety in the inherited bleeding disorders community. Biomedical and pharmaceutical…

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uniQure Announces Published Trial Results for Approved Gene Therapy

Industry News & Research

These results were based on the multi-year HOPE-B Trial program. uniQure recently announced publication of the clinical trial results associated with approval…

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New Medscape Activity to Help Institutions Offer Gene Therapy

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

NHF is pleased to announce that its collaboration with Medscape continues with a new accredited educational activity designed to help healthcare providers…

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BioMarin Announces FDA’s Extended Review of Investigational Hemophilia Gene Therapy

Industry News & Research

ROCTAVIAN™ is currently in multiple ongoing clinical trials to observe its safety and efficacy in adult patients with severe hemophilia A. On…

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What You Need to Know About Hemochromatosis

Industry News & Research, Living with a Bleeding Disorder

This blood disorder affects more than 1 million Americans and has no cure, but there are effective treatments. Hemochromatosis, also known as…

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Study Suggests Significant Rates of Depression and Anxiety for Individuals with VWD

Industry News & Research

Findings from this study indicate that a relatively high proportion of participants with von Willebrand disease met the criteria for both depression…

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Pfizer Announces Trial Updates for Investigational Hemophilia Gene Therapy

Industry News & Research

A primary focus of the trial was to compare annualized bleeding rates associated with a single infusion of the investigational gene therapy…

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Takeda Announces Trial Results on Investigational Therapy for Ultra Rare Blood Disorder

Industry News & Research

TAK-755 is being investigated for the treatment of congenital thrombotic thrombocytopenic purpura. Takeda recently announced favorable results from a phase 3 study…

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Biomarin Provides Hemophilia A Clinical Development Program Updates

Industry News & Research

BioMarin recently shared a community update on their Hemophilia A Clinical Development Program for valoctocogene roxaparvovec, the company’s investigational gene therapy currently…

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WFH Gene Therapy Registry Goes Live

Industry News & Research, Living with a Bleeding Disorder

The new registry will help monitor the long-term safety and efficacy of hemophilia gene therapies in people around the world. The World…

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Medicaid and CHIP Continuous Enrollment Unwinding

Advocacy & Legislation, Industry News & Research

Get information on your state’s plan to restart yearly Medicaid and Children’s Health Insurance Program (CHIP) eligibility reviews. Do you or a…

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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

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

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

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

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

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

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

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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