News

Pivotal data from the Phase 3 XTEND-Kids study evaluating ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] once-weekly prophylaxis, a factor VIII replacement…

BioMarin Pharmaceutical Inc. today announced the U.S. Food and Drug Administration approved ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults…

After several decades of preclinical and clinical research, pitfalls and progress, gene therapies for hemophilia A and B have become a reality…

that the call for applications for the Susan Skinner Memorial Fund (SSMF) Scholarship program is now open! Please spread the word among…

Early data on Medicaid “unwinding” shows an alarming rate of coverage losses for enrollees who may still be eligible. Colorado and Texas…

Authors of the review posit that reference intervals such as those utilized in thrombosis and hemostasis, are based on structural inequities. A…

HEMGENIX is indicated for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or…

Pfizer Inc. today announced their Phase 3 BASIS clinical trial evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and…

Hemophilia Federation of America has launched a new resource and search feature for clinical trials for patients with bleeding disorders.  “Patients and families have asked for…

HEMGENIX®, the first and only FDA approved gene therapy for hemophilia B,has been proven to elevate and sustain factor IX levels for…

Dr. Leanord Valentino announces his impending retirement. His statement reflects on the progress and initiatives made during his three and a half…

On May 11, 2023, the U.S. Food and Drug Administration released final guidance providing recommendations for evaluating blood and plasma donor eligibility using individual risk-based…

Marstacimab is a laboratory-engineered monoclonal antibody developed to treat hemophilia A and B patients, with or without inhibitors. Pfizer has announced positive…

The review provides a comprehensive overview of the persistent challenges and current mitigation strategies related to treating and managing hemophilia patients with…

On May 11, 2023, the U.S. Food and Drug Administration released final guidance providing recommendations for evaluating blood and plasma donor eligibility using individual risk-based…

Marstacimab is a laboratory-engineered monoclonal antibody developed to treat hemophilia A and B patients, with or without inhibitors. Pfizer has announced positive…

Female hemophilia A patients and carriers have more frequent bleeds than males, yet a smaller proportion receive factor to prevent or stop them, a U.S….

Are you a male, 18-29 years old, and on a prophy regimen for hemophilia treatment? You may qualify for a new program!…

The new guidelines cover recommendations for physical therapy, gene therapy readiness, vaccine guidance, and treatment. Download here. Source: National Hemophilia Foundation

Learn how this project from NHF, the American Thrombosis and Hemostasis Network, Bloodworks Northwest, and Bioverativ/Sanofi helped inform people with hemophilia on…

Between 2013-2017, the “My Life Our Future” (MLOF) project offered eligible individuals with hemophilia free genotyping, which is historically hard to access,…

As you may have heard, the Medicaid and CHIP (called Badgercare in Wisconsin) programs are re-starting eligibility reviews. Community members may receive…

The company received a Complete Response Letter from the FDA seeking more information relevant to their BLA for concizumab. Novo Nordisk has…

Desmopressin has the capacity to boost both factor VIII and von Willebrand factor levels. In July 2020 Ferring Pharmaceuticals suspended manufacturing of…