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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) aged 16 and older, 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).
VOXZOGO® is indicated for the treatment of achondroplasia in patients 2 years of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.
Strategically focusing on rare diseases with defined genetic defects, BioMarin strives to develop innovative therapies that are first- or best-in-class.
For each disease state we seek to treat, there are several organisations and resources that provide great insight and support services.
We provide a variety of opportunities to further support patient communities.
We provide product and funding support for independent research in rare diseases and genetic disorders to unleash scientific innovation and meet the unmet medical needs.
We seek to improve patient care and drive advancement in medical science, through providing qualified scientific researchers access to de-identified patient-level data collected in clinical trials.
We offer funding to support accredited or non-accredited medical education activities for HCPs regarding BioMarin-controlled or -influenced content.
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Patients and consumers should speak to their community pharmacy or prescriber for more information.
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