5 products found
BioMarin Products
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PALYNZIQ - Pegvaliase-Pqpz Injection for Phenylketonuria PKU
PALYNZIQ (pegvaliase-pqpz) Injection is the first FDA-approved enzyme substitution therapy for adults with PKU (phenylketonuria) who have uncontrolled blood Phe (phenylalanine) levels above 600 micromol/L (10 mg/dL) on their current treatment. PALYNZIQ is a once-daily self-administered therapy that acts independently of the phenylalanine hydroxylase (PAH) enzyme, so it is an option for all eligible adult patients living with ...
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Brineura - Cerliponase Alfa for Neuronal Ceroid Lipofuscinosis Type 2 CLN2 Disease
The first approved treatment for any form of Batten disease. Brineura (cerliponase alfa) is indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Brineura is the first enzyme replacement therapy to be directly administered into the fluid of the brain, treating the underlying cause of CLN2 ...
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Vimizim - Model MPS IVA - Elosulfase Alfa for Morquio A Syndrome
Vimizim (elosulfase alfa) is the first approved enzyme replacement therapy designed to address the underlying cause of Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA) — a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). VIMIZIM works at a cellular level to help with deficient enzyme ...
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Kuvan - Sapropterin Dihydrochloride for PKU
Kuvan® (sapropterin dihydrochloride) Tablets and Powder for Oral Solution is the first FDA-approved medication for phenylketonuria (PKU). Kuvan is a form of BH4, the cofactor of the PAH enzyme, which helps the enzyme break down Phe. Kuvan is to be used in conjunction with a Phe-restricted ...
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Aldurazyme - Aldurazyme - Laronidase for MPS I
ALDURAZYME (laronidase) is indicated for patients with Hurler and Hurler-Scheie forms of mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms. The risks and benefits of treating mildly affected patients with the Scheie form have not been established. ALDURAZYME has been shown to improve pulmonary function and walking capacity. ALDURAZYME has not been evaluated for effects on the central nervous system ...