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Brineura® is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.
Kuvan® is indicated for the treatment of hyperphenylalaninemia (HPA) by reducing blood phenylalanine levels in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.
NAGLAZYME® is indicated for the treatment of Mucopolysaccharidosis VI (MPS VI) storage disorder, a disease in which the enzyme level of Nacetylgalactosamine 4-sulfatase is absent or lower than normal.
PALYNZIQ™ is indicated for the treatment of patients with phenylketonuria (PKU) whose blood phenylalanine levels cannot be adequately controlled by other means such as by diet.
VIMIZIM® is indicated for the treatment of MPS IVA (Mucopolysaccharidosis Type IVA, Morquio A Syndrome).
Strategically focusing on rare diseases with defined genetic defects, BioMarin strives to develop innovative therapies that are first- or best-in-class.
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Patients and consumers should speak to their community pharmacy or prescriber for more information.