The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.
该发明涉及对
葡萄糖酰胺合成酶(GCS)的
抑制剂,可用于治疗代谢性疾病,如溶酶体贮存病,单独使用或与酶替代疗法联合使用,以及用于治疗癌症。