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Overview

BioMarin Pharmaceutical Inc. (BioMarin) is a biotechnology company, which is engaged in developing and commercializing therapies that address the root cause of genetic conditions. BioMarin's commercial products include Vimizim (elosulfase alpha), Naglazyme (galsulfase), Palynziq (pegvaliase-pqpz), Brineura (cerliponase alfa), Aldurazyme (laronidase), Roctavian (valoctocogene roxaparvovec), Kuvan (sapropterin dihydrochloride), and Voxzogo (vosoritide). Vimizim is an enzyme replacement therapy for the treatment of MPS IVA, a lysosomal storage disorder. The Roctavian is an adeno associated virus (AAV5) vector gene therapy designed to restore factor VIII plasma concentrations in patients with severe hemophilia A. Voxzogo is a once daily injection analog of C-type Natriuretic Peptide (CNP) for the treatment of achondroplasia. Brineura is a recombinant human tripeptidyl peptidase 1 (TPP1) for the treatment of patients with CLN2, a form of Batten disease.