BioMarin announces NEJM publishes 3 years of follow-up data in phase 1/2 study of valoctocogene roxaparvovec gene therapy for hemophilia A
BioMarin Pharmaceutical Inc, a global biotechnology company announced that the New England Journal of Medicine (NEJM) published an independently peer-reviewed article on up to three years of data from an ongoing phase 1/2 study to evaluate safety and efficacy of investigational AAV gene therapy, valoctocogene roxaparvovec, for severe hemophilia A. This is the second article published by the NEJM on valoctocogene roxaparvovec.