naglazyme 5mg5ml solution for infusion vials
biomarin europe ltd - galsulfase - solution for infusion - 1mg/1ml
kuvan 100mg soluble tablets
biomarin europe ltd - sapropterin dihydrochloride - soluble tablet - 100mg
firdapse 10mg tablets
biomarin europe ltd - amifampridine phosphate - tablet - 10mg
vimizim 5mg5ml concentrate for solution for infusion vials
biomarin europe ltd - elosulfase alfa - solution for infusion - 1mg/1ml
brineura 150mg5ml solution for infusion vials
biomarin europe ltd - cerliponase alfa - solution for infusion - 30mg/1ml
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 cerliponase alfa (rch) 150 mg/5 ml solution for injection vial with flushing solution vial
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.
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
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.
palynziq- pegvaliase-pqpz injection, solution
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).