公开(公告)号：US11040087B2

公开(公告)日：2021-06-22

申请号：US16324194

申请日：2017-08-18

申请人： NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY ,  TOKYO METROPOLITAN GERIATRIC HOSPITAL AND INSTITUTE OF GERONTOLOGY ,  OSAKA PREFECTURAL HOSPITAL ORGANIZATION

发明人： Tatsushi Toda ,  Kazuhiro Kobayashi ,  Motoi Kanagawa ,  Tamao Endo ,  Hiroshi Manya ,  Yoshinao Wada ,  Michiko Tajiri

IPC分类号： A61K38/45 ,  A61P21/00 ,  G01N33/68

摘要： Provided is a therapeutic agent that effectively acts on a disease associated with abnormal glycosylation of dystroglycan. Also provided is a testing method for diseases associated with abnormal glycosylation of dystroglycan. Specifically, provided is a therapeutic agent for diseases associated with abnormal glycosylation of dystroglycan, containing CDP-ribitol as an active ingredient. Ribitol-phosphate is important in the glycan structure of dystroglycan. In order for ribitol-phosphate to be incorporated into a dystroglycan glycan, a material therefor (sugar donor) is required. In the present invention, it has been found for the first time that CDP-ribitol serves as the sugar donor. It has been confirmed that the glycan of ISPD-deficient cells can be restored by administering CDP-ribitol. Thus, the present invention, which allows CDP-ribitol to be utilized for supplementation therapy, has been completed.