News

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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Guilt in Mothers of Children with Hemophilia Focus of New Study

Health and Well Being, Living with a Bleeding Disorder

Study authors looked at potential contributing factors to guilt, coping strategies, and perception of children’s life satisfaction. Researchers from the Children’s Healthcare…

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Start the Conversation: From Patient to Physician

Health and Well Being, Living with a Bleeding Disorder

To end Bleeding Disorders Awareness Month and celebrate National Doctors Day, Dr. Aaron Chen shares his story of how living with thalassemia…

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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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New Survey Spotlights Ongoing Concerns for People with Hemophilia B

Health and Well Being, 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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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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GLHF’s Wisconsin Bleeding Disorders Conference is June 2-4. Registration is Open Until April 24!

GLHF News

Each year Great Lakes Hemophilia Foundation hosts the Wisconsin Bleeding Disorders Conference (WBDC) for individuals and families living with a bleeding disorder….

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Is your Camper Ready to Move into a Leadership Role? Applications for Camp Klotty Pine’s LIT Program are Due March 26. Join us April 29-30 for our LIT Retreat.

GLHF News

Calling all former Camp Klotty Pine campers and Leaders-in-Training (LITs)! Is your camper age 15-18 and ready to move into a leadership…

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Calling all Campers! Get Ready to Join us August 7-12.

GLHF News

Join us for Camp Klotty Pine, August 7-12. Camp Klotty Pine creates a magical, life-changing experiences for children living with a bleeding disorder….

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Advocates Met with State Reps for WI Legislative Day – All Copays Count Legislation is Top Priority

Advocacy & Legislation, GLHF News

Community advocates gathered on March 1st at the State Capitol for GLHF’s Wisconsin Legislative Day. We shared stories of life with a bleeding…

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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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