PREVENTIVE AND THERAPEUTIC AGENT FOR LYSOSOMAL DISEASES
|Appl. No. (filing date)||PCT/JP2019/004766 (2019/02/05)|
|Pub. No. (Pub. date)||WO2019/156252 (2019/08/15)|
|Priority claim No. (Priority date)||
|Inventor||SAKURAI, Hidetoshi, etc.|
|Title of Invention||PREVENTIVE AND THERAPEUTIC AGENT FOR LYSOSOMAL DISEASES|
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.