News
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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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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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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…