BioMarin

Naglazyme (galsulfase) is used to treat mucopolysaccharidosis VI (MPS VI), a rare metabolic disorder. Vimizim (elosulfase alfa) - To treat Morquio A syndrome (MPS IVA), a genetic disorder that affects bone development. Kuvan (sapropterin dihydrochloride) - Approved for the treatment of Phenylketonuria (PKU), a metabolic disorder that affects the body's ability to break down phenylalanine. Brineura (cerliponase alfa) - Treat Neuronal Ceroid Lipofuscinosis Type 2 (CLN2), a rare neurodegenerative disorder in children. Palynziq (pegvaliase-pqpz) - Approved for treating Phenylketonuria (PKU) in adults with uncontrolled blood phenylalanine levels. Firdapse (amifampridine) - Approved for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), a rare autoimmune disorder affecting neuromuscular transmission.