News

Summit Emphasizes Pharmacovigilance in the Inherited Bleeding Disorders Community
Uncategorized
The NHF/HFA Safety Summit yielded a series of recommendations related to product safety in the inherited bleeding disorders community. Biomedical and pharmaceutical…

uniQure Announces Published Trial Results for Approved Gene Therapy
Industry News & Research, Uncategorized
These results were based on the multi-year HOPE-B Trial program. uniQure recently announced publication of the clinical trial results associated with approval…

New Medscape Activity to Help Institutions Offer Gene Therapy
Industry News & Research, Uncategorized
NHF is pleased to announce that its collaboration with Medscape continues with a new accredited educational activity designed to help healthcare providers…

BioMarin Announces FDA’s Extended Review of Investigational Hemophilia Gene Therapy
Industry News & Research, Uncategorized
ROCTAVIAN™ is currently in multiple ongoing clinical trials to observe its safety and efficacy in adult patients with severe hemophilia A. On…

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…

What You Need to Know About Hemochromatosis
Industry News & Research, Living with a Bleeding Disorder, Uncategorized
This blood disorder affects more than 1 million Americans and has no cure, but there are effective treatments. Hemochromatosis, also known as…

GLHF’s Wisconsin Bleeding Disorders Conference is June 2-4. Registration Opens this Month!
GLHF News, Uncategorized
Each year Great Lakes Hemophilia Foundation hosts the Wisconsin Bleeding Disorders Conference (WBDC) for individuals and families living with a bleeding disorder….

Join us for a Bloody Good Time as GLHF’s Bloody Mary Competitions Continue this Spring in Madison and Green Bay. Drink Local. Give Local. Get Your Tickets Today!
GLHF News, Uncategorized
Madison’s Best Bloody is April 16, and Green Bay’s Best Bloody is May 21. Get your tickets today! Milwaukee’s Best Bloody, held…

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, Uncategorized
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…

Calling all Campers! Camp Klotty Pine is August 7-12. Join us for our Camp Open House on April 30.
GLHF News, Uncategorized
Camp Klotty Pine creates magical, life-changing experiences for children living with a bleeding disorder. At camp, children develop confidence, independence and build…

Advocates Met with State Reps for WI Legislative Day – All Copays Count Legislation is Top Priority
Advocacy & Legislation, GLHF News, Uncategorized
Community advocates gathered on March 1st at the State Capitol for GLHF’s Wisconsin Legislative Day. We shared stories of life with a bleeding…

Study Suggests Significant Rates of Depression and Anxiety for Individuals with VWD
Industry News & Research, Uncategorized
Findings from this study indicate that a relatively high proportion of participants with von Willebrand disease met the criteria for both depression…

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…

Takeda Announces Trial Results on Investigational Therapy for Ultra Rare Blood Disorder
Industry News & Research, Uncategorized
TAK-755 is being investigated for the treatment of congenital thrombotic thrombocytopenic purpura. Takeda recently announced favorable results from a phase 3 study…

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

WFH Gene Therapy Registry Goes Live
Uncategorized
The new registry will help monitor the long-term safety and efficacy of hemophilia gene therapies in people around the world. The World…

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…

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…

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…

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….

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…

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…

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….

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…