BioMarin Pharmaceutical Inc. develops and commercializes pharmaceuticals for serious diseases and medical conditions in the United States, Europe, Latin America, and internationally. Its commercial products include Aldurazyme used for the treatment of patients with mucopolysaccharidosis I, a genetic disease; Kuvan, a proprietary synthetic oral form of 6R-BH4 used to treat patients with phenylketonuria (PKU), an inherited metabolic disease; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with mucopolysaccharidosis VI; and Vimizim, an enzyme replacement therapy for the treatment of MPS IV A, a lysosomal storage disorder. The company also conducts clinical trials on several investigational product candidates for the treatment of various diseases, including Brineura for the treatment of late infantile neuronal ceroid lipofuscinosis, a form of Batten disease; pegvaliase, an enzyme substitution therapy for the treatment of PKU; vosoritide, a peptide therapeutic for the treatment of achondroplasia; BMN 270, an AAV VIII vector and Factor VIII gene therapy drug development candidate, for the treatment of hemophilia A; and BMN 250, a novel fusion of alpha-N-acetyglucosaminidase for the treatment of Sanfilippo B syndrome, or mucopolysaccharidosis type IIIB. The company serves specialty pharmacies and end-users, such as hospitals and foreign government agencies; and distributors and pharmaceutical wholesalers. BioMarin Pharmaceutical Inc. has a collaboration agreement with Genzyme Corporation. The company was founded in 1996 and is headquartered in San Rafael, California.
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