The invention relates to inhibitors of glucosylceramide synthase (GCS) 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.
该发明涉及抑制
葡萄糖鞘氨醇合成酶(GCS)的
抑制剂,用于治疗代谢性疾病,如溶酶体贮积病,单独或与酶替代疗法、囊性疾病以及癌症的联合治疗。