naglazyme
biomarin international limited - galsulfase - mucopolysaccharidosis vi - other alimentary tract and metabolism products, - naglazyme is indicated for long-term enzyme-replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis vi (mps vi; n-acetylgalactosamine-4-sulfatase deficiency; maroteaux-lamy syndrome) (see section 5.1). as for all lysosomal genetic disorders, it is of primary importance, especially in severe forms, to initiate treatment as early as possible, before appearance of non-reversible clinical manifestations of the disease. a key issue is to treat young patients aged
vimizim
biomarin international limited - recombinant human n-acetylgalactosamine-6-sulfatase - mucopolysaccharidosis iv - other alimentary tract and metabolism products, - vimizim is indicated for the treatment of mucopolysaccharidosis, type iva (morquio a syndrome, mps iva) in patients of all ages.
kuvan
biomarin international limited - sapropterin dihydrochloride - phenylketonurias - other alimentary tract and metabolism products, - kuvan is indicated for the treatment of hyperphenylalaninaemia (hpa) in adults and paediatric patients of all ages with phenylketonuria (pku) who have been shown to be responsive to such treatment.kuvan is also indicated for the treatment of hyperphenylalaninaemia (hpa) in adults and paediatric patients of all ages with tetrahydrobiopterin (bh4) deficiency who have been shown to be responsive to such treatment.
roctavian
biomarin international limited - valoctocogene roxaparvovec - antihemorrhagics - treatment of severe haemophilia a (congenital factor viii deficiency) in adult patients without a history of factor viii inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (aav5).
brineura
biomarin international limited - cerliponase alfa - neuronal ceroid-lipofuscinoses - other alimentary tract and metabolism products, - brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (cln2) disease, also known as tripeptidyl peptidase 1 (tpp1) deficiency,
palynziq
biomarin international limited - pegvaliase - phenylketonurias - other alimentary tract and metabolism products - palynziq is indicated for the treatment of patients with phenylketonuria (pku) aged 16 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite prior management with available treatment options.
voxzogo
biomarin international limited - vosoritide - achondroplasia - drugs for treatment of bone diseases - voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. the diagnosis of achondroplasia should be confirmed by appropriate genetic testing.
palynziq solution
biomarin international limited - pegvaliase - solution - 2.5mg - pegvaliase 2.5mg
palynziq solution
biomarin international limited - pegvaliase - solution - 10mg - pegvaliase 10mg
palynziq solution
biomarin international limited - pegvaliase - solution - 20mg - pegvaliase 20mg