BioMarin Pharmaceutical Inc(BM8)

Description: BioMarin Pharmaceutical Inc. engages in the development and commercialization of therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; 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 Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops Roctavian, an adeno associated virus vector, for the treatment of patients with severe hemophilia A. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. It has license and collaboration agreements with Sarepta Therapeutics; Ares Trading S.A.; Catalyst Pharmaceutical Partners, Inc.; and CAMP4 Therapeutics Corporation. The company was incorporated in 1996 and is headquartered in San Rafael, California.

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Keywords: Rare Diseases Virotherapy Orphan Drug Metabolic Disease Lipid Storage Disorders Phenylketonuria Pharmaceutical Wholesalers Enzyme Replacement Therapy Mucopolysaccharidosis Pegylation Biomarin Pharmaceutical Lysosomal Storage Disorder Iduronidase Achondroplasia Naglazyme Severe Hemophilia A Aldurazyme Batten Disease Serious And Life Threatening Rare Diseases Tetrahydrobiopterin

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Home Page: www.biomarin.com

770 Lindaro Street
San Rafael, CA 94901
United States
Phone: 415 506 6700

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Officers

Exchange: F

Country: DE : Germany

Currency: Euro (€)