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BMN 250

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Company BioMarin Pharmaceutical Inc.
DescriptionN-acetylglucosaminidase alpha (NAGLU) fused with a peptide derived from insulin-like growth factor-2 (IGF-2)
Molecular Target N-acetylglucosaminidase alpha (NAGLU)
Mechanism of ActionEnzyme replacement therapy
Therapeutic ModalityBiologic: Fusion protein
Latest Stage of DevelopmentPhase I/II
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo type B syndrome)
Regulatory Designation U.S. - Orphan Drug (Treat mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo type B syndrome))

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