申请人:Genzyme Corporation
公开号:US20140371460A1
公开(公告)日:2014-12-18
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.
本发明涉及一种对糖脂合成酶(GCS)的抑制剂,用于治疗代谢性疾病,例如溶酶体贮存病,可单独使用或与酶替代治疗相结合,并用于治疗癌症。