BioMarin Pharmaceutical Inc.
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BioMarin Pharmaceutical Inc. (BioMarin) is a biotechnology company that develops and commercializes therapies that address the root cause of genetic conditions. The Company's portfolio consists of seven commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. BioMarin's commercial products include Vimizim (elosulfase alpha), Naglazyme (galsulfase), Kuvan (sapropterin dihydrochloride), Palynziq (pegvaliase-pqpz), Brineura (cerliponase alfa), Roctavian (valoctocogene roxaparvovec) and Voxzogo (vosoritide). The Roctavian is an adeno associated virus (AAV5) vector drug development candidate designed to restore factor VIII plasma concentrations in patients with severe hemophilia A. The Company’s clinical development programs include BMN 331, an AAV5 mediated gene therapy for people living with Hereditary Angioedema (HAE) and BMN 255 is a small-molecule therapy that is designed to treat hyperoxaluria in chronic liver disease.
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