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公开(公告)号:US20220409595A1
公开(公告)日:2022-12-29
申请号:US17824552
申请日:2022-05-25
Applicant: GENZYME CORPORATION
Inventor: John P. LEONARD , Jean-François DELEUZE , Jean-Michel ITIER , Cécile ORSINI , Gwénaëlle RET-LECUELLE , Sandra VIALE
IPC: A61K31/439
Abstract: The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy.