Patent Portfolio

PREVENTIVE AND THERAPEUTIC AGENT FOR LYSOSOMAL DISEASES

Ref. No. AJ198
Appl. No.
（filing date）		 PCT/JP2019/004766（2019/2/5）
Pub. No.
（Pub. date）		 WO2019/156252（2019/8/15）
Priority claim No.
（Priority date）

JP2018-019698 (2018/02/06)
Patent No.

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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

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Updated date 2020/2/28
Information
(External link)		 https://www.j-platpat.inpit.go.jp/c1800/PU/WO-A-2019-156252/FA1A7440A68C972DA12C2ED217E6152A72C6D8C7B16F6984C8CF660449B860E6/50/en
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