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Company Amicus Therapeutics Inc.
DescriptionNext-generation small molecule chaperone that binds to glucocerebrosidase (GBA; GCase)
Molecular Target Glucocerebrosidase (GBA) (GCase)
Mechanism of Action 
Therapeutic ModalitySmall molecule

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Disease CategoryStandard IndicationIndication DetailsPartnersMilestonesLatest Stage of DevelopmentRegulatory Designation
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