United Kingdom - English - MHRA (Medicines & Healthcare Products Regulatory Agency)

biomarin europe ltd - galsulfase - solution for infusion - 1mg/1ml

United Kingdom - English - MHRA (Medicines & Healthcare Products Regulatory Agency)

biomarin europe ltd - sapropterin dihydrochloride - soluble tablet - 100mg

United Kingdom - English - MHRA (Medicines & Healthcare Products Regulatory Agency)

biomarin europe ltd - amifampridine phosphate - tablet - 10mg

United Kingdom - English - MHRA (Medicines & Healthcare Products Regulatory Agency)

biomarin europe ltd - elosulfase alfa - solution for infusion - 1mg/1ml

United Kingdom - English - MHRA (Medicines & Healthcare Products Regulatory Agency)

biomarin europe ltd - cerliponase alfa - solution for infusion - 30mg/1ml

European Union - English - EMA (European Medicines Agency)

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).

Australia - English - Department of Health (Therapeutic Goods Administration)

biomarin pharmaceutical australia pty ltd - cerliponase alfa, quantity: 30 mg/ml - injection, solution - excipient ingredients: dibasic sodium phosphate heptahydrate; sodium chloride; potassium chloride; magnesium chloride hexahydrate; calcium chloride dihydrate; water for injections; monobasic sodium phosphate monohydrate - brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (cln2) disease, also known as tripeptidyl peptidase 1 (tpp1) deficiency.

European Union - English - EMA (European Medicines Agency)

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

Israel - English - Ministry of Health

medison pharma ltd - elosulfase alfa - concentrate for solution for infusion - elosulfase alfa 1 mg/ml - elosulfase alfa - vimizim is indicated for the treatment of mucopolysaccharidosis, type iva (morquio a syndrome ,mps iva) in patients of all ages.

United States - English - NLM (National Library of Medicine)

biomarin pharmaceutical inc. - pegvaliase (unii: n6uah27euv) (pegvaliase - unii:n6uah27euv) - palynziq is indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria (pku) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/l on existing management. none. risk summary based on findings in studies of pregnant animals without pku treated with pegvaliase-pqpz, palynziq may cause fetal harm when administered to a pregnant woman. limited available data with pegvaliase-pqpz use in pregnant women are insufficient to inform a drug-associated risk of adverse developmental outcomes. there are risks to the fetus associated with poorly controlled phenylalanine concentrations in women with pku during pregnancy including increased risk for miscarriage, major birth defects (including microcephaly, major cardiac malformations), intrauterine fetal growth retardation, and future intellectual disability with low iq; therefore, phenylalanine concentrations should be closely monitored in women with pku during pregnancy (see clinical considerations and data).