• 专利标题:   Pharmaceutical composition useful for preventing or treating lysosomal storage disease caused by substrate accumulated in lysosome e.g. Fabry disease by inhibiting accumulation of substrate in lysosome, comprises graphene quantum dots.
  • 专利号:   WO2020159159-A2, KR2020094320-A, WO2020159159-A3, KR2179430-B1
  • 发明人:   KANG K, HONG B H, KANG I, YOO J M, KIM D, SUN K, KANG I S, MIN Y J, HOON K D
  • 专利权人:   BIOGRAPHENE INC, UNIV SEOUL NAT R DB FOUND, BIO GRAPHENE INC
  • 国际专利分类:   A61K033/44, A61K009/14, A61P043/00
  • 专利详细信息:   WO2020159159-A2 06 Aug 2020 A61K-033/44 202069 Pages: 30
  • 申请详细信息:   WO2020159159-A2 WOKR001162 23 Jan 2020
  • 优先权号:   KR011642

▎ 摘  要

NOVELTY - Pharmaceutical composition comprises graphene quantum dots as an active ingredient. USE - The pharmaceutical composition is useful for preventing or treating lysosomal storage disease caused by a substrate accumulated in lysosome, inhibiting accumulation of substrate in lysosome and decomposing previously accumulated substrate in an individual. The graphene quantum dots are used for preparing a drug for preventing or treating lysosomal storage disease. The lysosome accumulation disease is chosen from pompe disease, Gauche disease, Fabry disease, GM1-ganglioside accumulation disease, Tay-Sachs disease, Sandhoff disease, Krabbe disease, Farber disease, otitis media, dystrophy, Hurler-Scheie syndrome, Hunter's disease, Sanfilippo syndrome, San Philip's syndrome B, San Philip's syndrome C, San Philip's syndrome D, Morquio's disease A, morquio B syndrome, Maroteaux-Lamy syndrome, sly disease, alpha -mannosidosis, beta -mannosidosis, fucosidosis, sialidosis, Schindler-Kanza disease, and Niemann-Pick disease (all claimed).