Brineura (Cerliponase Alfa)
Brineura 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…
BioMarin Pharmaceutical Inc. Products: Prescription Medications Section
Kuvan (SAPROPTERIN DIHYDROCHLORIDE)
Kuvan is indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-…
NAGLAZYME (Galsulfase)
NAGLAZYME is indicated for patients with Mucopolysaccharidosis VI (MPS VI. Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity. NAGLAZYME is…
Palynziq (Pegvaliase-pqpz)
Palynziq is indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600…
ROCTAVIAN (Valoctocogene Roxaparvovec-rvox)
ROCTAVIAN is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII…