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 Vimizim, an enzyme replacement therapy for the treatment of MPS IV A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with mucopolysaccharidosis VI; Kuvan, a proprietary synthetic oral form of 6R-BH4 used to treat patients with phenylketonuria (PKU), an inherited metabolic disease; Aldurazyme used for the treatment of patients with mucopolysaccharidosis I, a genetic disease; Firdapse, a form of 3,4-diaminopyridine used for the treatment of Lambert Myasthenic Syndrome, an autoimmune disease. The company also conducts clinical trials on several investigational product candidates for the treatment of various diseases, including drisapersen, an exon-51 skipping compound for the treatment of Duchenne muscular dystrophy (DMD); pegvaliase, an enzyme substitution therapy for the treatment of PKU; reveglucosidase alfa, an enzyme replacement therapy for Pompe disease; talazoparib, an orally available poly-ADP ribose polymerase inhibitor for cancer treatment; BMN 111, a peptide therapeutic for achondroplasia treatment; BMN 044, BMN 045, and BMN 053 for DMD treatment; and cerliponase alfa for the treatment of late infantile neuronal ceroid lipofuscinosis. In addition, it develops preclinical product candidates for genetic and other metabolic diseases, such as BMN 270 and BMN 250. The company serves specialty pharmacies and end-users, such as hospitals and foreign government agencies; and distributors and pharmaceutical wholesalers. S.A. BioMarin Pharmaceutical Inc. has a collaboration agreement with Genzyme Corporation; and an agreement with Merck Serono. The company was founded in 1996 and is headquartered in San Rafael, California.