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Plicera, afegostat, isofagomine tartrate (AT2101)

Also known as: afegostat tartrate

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Company Amicus Therapeutics Inc.
DescriptionOrally available small molecule chaperone that binds to glucocerebrosidase
Molecular Target Glucocerebrosidase (GBA) (GCase)
Mechanism of ActionGlucocerebrosidase (GBA) destabilization
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPreclinical
Standard IndicationGaucher's disease
Indication DetailsTreat Gaucher's disease; Treat Type I Gaucher's disease
Regulatory Designation

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