Skip to main content


Gene Therapy

BioMarin has published results of its Phase III clinical trials of its investigational gene therapy, intended for the treatment of adults with severe hemophilia A. The trial results of valoctocogene roxaparvovec was published in the New England Journal of Medicine on March 17, 2022.
In the published results, BioMarin shared that after a single infusion of valoctocogene roxaparvovec, patients experienced substantially reduced annualized bleeding rates, reduced Factor VIII utilization, and increased Factor VIII activity. Valoctocogene roxaparvovec has been studied longer than any other gene therapy for hemophilia A.

Von Willebrand’s Disease

The American Society of Hematology and the International Society on Thrombosis and Haemostasis, the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) are endorsing new guidelines regarding the diagnosis of von Willebrand’s disease. Key recommendations of these guidelines include the role of bleeding-assessment tools in the assessment of patients suspected of VWD, diagnostic assays and laboratory cutoffs for type 1 and type 2 VWD, how to approach a type 1 VWD patient with normalized levels over time, and the role of genetic testing vs phenotypic assays.