News

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…

These updates were presented during the International Society on Thrombosis and Haemostasis (ISTH) Congress in London. Sanofi recently shared new clinical trial…

The European Commission granted conditional approval to BioMarin’s Hemophilia A gene therapy Valoctocogene Roxaparvovec which will be known as Roctavian. With this…

In early October, NHF joined members of The All Copays Count Coalition (ACCC), sending a letter signed by 73 leading patient advocacy and…

This trial is evaluating the efficacy and safety of a single infusion of an investigational gene therapy in individuals with moderately severe…

Biomarin recently provided an “Update for the Hemophilia Community,” on the ongoing clinical trial program for its investigational hemophilia A gene therapy,…

HFA and 25 other patient groups issued the following statement in response to Wednesday’s court ruling in Braidwood v. Becerra (a lawsuit formerly known…

Initial data from the survey were presented at the recent Thrombosis and Hemostasis Summit of North America and NHF’s Bleeding Disorders Conference…

BIVV001 is a recombinant factor VIII therapy (rFVIII) developed for the prevention of bleeding episodes in hemophilia A patients via once weekly…

In this video, Dr. Bethany Samuelson Bannow, of Oregon Health & Science University, discusses the role of iron in the body, the…

Gene Therapy is definitely a dominant topic in the bleeding disorders community, with new articles being published regularly. Read some of the…

by: Laurie Kelley Thoughts on how the Supreme Court decision on abortion might affect genetic testing outcomes. Read more here. The recent…

Solid market position of Hemlibra and strong prescriber interest in RNAi and TFPI treatment options, combined with patient concerns regarding gene therapies,…

The National Hemophilia Foundation’s 16th Workshop on Novel Technologies and Gene Transfer for Hemophilia, held in November, addressed these questions and more….

Current hemophilia B treatments offer strong protection from bleeds, but impact patient lifestyle and require consistent infusions to keep protection levels high.1…