申请人:Genzyme Corporation
公开号:US11008316B2
公开(公告)日:2021-05-18
The invention relates to inhibitors of glucosylceramide synthase (GCS), such as Compound of Formula I, shown below, as defined herein, useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.
本发明涉及葡萄糖酰胺合成酶(GCS)的抑制剂,例如下图所示的式 I 化合物,如本文所定义的,可用于治疗代谢性疾病,如溶酶体贮积症,可单独使用或与酶替代疗法、囊性疾病联合使用,也可用于治疗癌症。