BioMarin Pharmaceutical


Focus: Child Neurology - late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) and other rare genetic diseases

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. Approved products include the first and only therapies for PKU, LEMS, MPS I, MPS VI, MPS IVA, and CLN2 disease. Clinical development programs include investigational therapies for Hemophilia A, Achondroplasia, MPS IIIB, Friedreich’s Ataxia and other rare diseases.  Please visit to learn more.