News
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…
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…
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…
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…
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
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
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…
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…
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
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
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
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
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
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
by: Donna Behen Blood Safety Millions of Americans who have or are at high risk for heart disease regularly take anti-platelet drugs…
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…
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,…
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…
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…
FDA Approves First Gene Therapy to Treat Adults with Hemophilia B
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
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…
Sanofi Provides Fitusiran Trial Updates at ISTH
Industry News & Research
These updates were presented during the International Society on Thrombosis and Haemostasis (ISTH) Congress in London. Sanofi recently shared new clinical trial…
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…
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…