PREVENTIVE AND THERAPEUTIC AGENT FOR LYSOSOMAL DISEASES
| Ref. No. | AJ198 |
|---|---|
| Appl. No. (filing date) | PCT/JP2019/004766 (2019/02/05) |
| Pub. No. (Pub. date) | WO2019/156252 (2019/08/15) |
| Priority claim No. (Priority date) | JP2018-019698 (2018/02/06) |
| Patent No. | - |
| Inventor | SAKURAI, Hidetoshi, etc. |
| Assignee | Kyoto University |
| Title of Invention | PREVENTIVE AND THERAPEUTIC AGENT FOR LYSOSOMAL DISEASES |
| Abstract | Provided is a lysosomal disease preventive and/or therapeutic agent comprising an HMG-CoA reductase inhibitor. The inventors found that HMG-CoA reductase inhibitors decreased autophagosome accumulation in a skeletal muscle cell differentiated from an iPS cell derived from patients of Pompe Disease and/or GNE Myopathy, and thereby completed this invention. |
| Comments | - |
| Updated date | 2020/02/28 |
| Publication |
