BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Aldurazyme to treat mucopolysaccharidosis I, a genetic disease; Brineura for the treatment of late infantile neuronal ceroid lipofuscinosis type 2, a form of Batten disease; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with mucopolysaccharidosis VI; Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme for adult patients with PKU; and Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis IV Type A, a lysosomal storage disorder. In addition, it develops valoctocogene roxaparvovec, an adeno associated virus vector, which is in Phase III clinical trial for the treatment of patients with severe hemophilia A; vosoritide that is in Phase III clinical trial for the treatment of achondroplasia, a form of disproportionate short stature in humans; BMN 307, an AAV5 mediated gene therapy, which is in phase 1/2 clinical trial to normalize blood Phe concentration levels in patients with PKU; BMN 331 for the treatment of hereditary angioedema; BMN 255 for the treatment of chronic renal disease; and BMN 351 for the treatment of Duchenne Muscular Dystrophy. The company has collaboration and license agreements with Sarepta Therapeutics, Asubio Pharma Co., Ltd., and DiNAQOR AG. The company serves specialty pharmacies, hospitals, and foreign government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. It has collaboration with Allen Institute. BioMarin Pharmaceutical Inc. was incorporated in 1996 and is headquartered in San Rafael, California.
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