News

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

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

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

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

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

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

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

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

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

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

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

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 CoalitionThe organization’s public policy representatives offer new perspectives in the mission for blood and…

This assay is designed to help determine factor IX activity levels in the plasma of individuals with hemophilia B who are two…

If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A PDUFA Target Action Date is March 31, 2023….

Pfizer had positive results from the Phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult…

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’s Judith Graham Pool Postdoctoral Research Fellowship program turns 50. We spoke with five awardees about how the fellowship shaped their careers….

by: Donna Behen Blood Safety Millions of Americans who have or are at high risk for heart disease regularly take anti-platelet drugs…

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’s Word from Washington outlines a massive year-end omnibus spending bill containing numerous important health provisions affecting Medicaid, telehealth,…

Pfizer and Sangamo Therapeutics announced that the Phase 3 AFFINE study evaluating giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately…

Five years ago, Hemophilia Federation of America articulated a broad vision that includes advocating for improved care and quality of life for…

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…