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公开(公告)号:US20250026762A1
公开(公告)日:2025-01-23
申请号:US18712270
申请日:2022-11-28
Applicant: Merck Sharp & Dohme LLC
Inventor: Mark E. Fraley , Tao Liang , H. Marie Loughran , Anthony J. Roecker , Kathy M. Schirripa , Ling Tong , Ashwin U. Rao
IPC: C07D487/08 , A61K31/551 , C07D519/00
Abstract: The present invention relates to Compounds of Formula (I) and pharmaceutically acceptable salts or prodrug thereof. The present invention also relates to compositions comprising at least one compound of Formula (I), and methods of using the compounds of Formula (I) for treatment or prophylaxis of lysosomal storage diseases, neurodegenerative disease, cystic disease, cancer, or a diseases or disorders associated with elevated levels of glucosylceramide (GlcCer), glucosylsphingosine (GlcSph) and/or other glucosylceramide-based glycosphingolipids (GSLs).