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BRINEURA® (cerliponase alfa)
KUVAN® (sapropterin dihydrochloride)
NAGLAZYME® (galsulfase (rch))
VIMIZIM® (elosulfase alfa (rch))
VOXZOGO® (vosoritide)
Achondroplasia
Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease
Maroteaux-Lamy syndrome (MPS VI)
Morquio A (MPS IVA)
Phenylketonuria (PKU)
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) in sapropterin-responsive adult and paediatric patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.
NAGLAZYME® is indicated as long term enzyme replacement therapy in patients with Mucopolysaccharidosis VI (MPS VI, Nacetylgalactosamine 4- sulfatase deficiency, Maroteaux-Lamy syndrome).
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.