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BMN 701 (formerly ZC-701)

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Collapse Summary General Information
Company BioMarin Pharmaceutical Inc.
DescriptionFusion of insulin-like growth factor-2 (IGF-2) and acid alpha glucosidase (GAA) that uses Glycosylation Independent Lysosomal Targeting (GILT) technology
Molecular Target Insulin-like growth factor-2 (IGF-2) receptor (IGF2R) (M6PR)
Mechanism of Action 
Therapeutic ModalityBiologic: Fusion protein
Latest Stage of DevelopmentPhase III
Standard IndicationPompe's disease
Indication DetailsTreat late-onset Pompe's disease; Treat Pompe's disease
Regulatory Designation

U.S. - Orphan Drug (Treat Pompe's disease)

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