[EN] MODIFIED ISOINDOLINONES AS GLUCOSYLCERAMIDE SYNTHASE INHIBITORS<br/>[FR] ISOINDOLINONES MODIFIÉES EN TANT QU'INHIBITEURS DE GLUCOSYLCÉRAMIDE SYNTHASE
申请人:MERCK SHARP & DOHME
公开号:WO2022115301A1
公开(公告)日:2022-06-02
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).
本发明涉及式(I)化合物及其药学上可接受的盐或前药。本发明还涉及包含至少一种式(I)化合物的组合物,以及使用式(I)化合物治疗或预防溶酶体贮积病、神经退行性疾病、囊性疾病、癌症或与葡糖鞘氨醇脂(GlcCer)、葡糖鞘氨醇(GlcSph)和/或其他葡糖鞘氨醇基糖脂(GSL)水平升高相关的疾病或障碍的方法。