- 专利标题： CALMODULIN INHIBITORS, CHK2 INHIBITORS AND RSK INHIBITORS FOR THE TREATMENT OF RIBOSOMAL DISORDERS AND RIBOSOMAPATHIES
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申请号： EP24174240.2申请日： 2017-07-13
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公开(公告)号： EP4427760A3公开(公告)日： 2024-11-13
- 发明人: BRADNER, James , QI, Jun , BUCKLEY, Dennis , ZON, Leonard I , MACARI, Elizabeth
- 申请人： Children's Medical Center, Corp. , Dana-Farber Cancer Institute
- 申请人地址： US Boston, MA 02115 55 Shattuck Street; US Boston, MA 02215 450 Brookline Avenue
- 代理机构： Purdylucey Intellectual Property
- 优先权： US201662361631 P 20160713
- 主分类号： A61K31/4375
- IPC分类号： A61K31/4375 ; A61K31/145 ; A61K31/4418 ; A61P7/06 ; A61K45/06 ; A61K31/4045 ; A61K31/519 ; A61K31/7048 ; A61K31/541
摘要：
The present invention relates to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathies, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to the use of novel classes of compounds, i.e. inhibitors of RSK (p90S6K); inhibitors of p70S6K; and inhibitors of rps6, to treat ribosomal disorders and ribosomopathies. In some embodiments, the invention relates to the use of specific Chk2 inhibitors and to the use of specific phenothiazine derivatives to treat ribosomal disorders and ribosomopathies, e.g. DBA.
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