发明名称 |
PROPHYLACTIC OR AMELIORATING AGENT FOR GENETIC DISEASES |
摘要 |
An object of the present invention is to provide a prophylactic or ameliorating agent for a genetic disease that is caused by a mutation in an exon of a gene and is capable of forming a functional truncated protein by skipping of the exon comprising the mutation. The prophylactic or ameliorating agent used in the present invention is a prophylactic or ameliorating agent for a genetic disease that is caused by a mutation in an exon of a gene and is capable of forming a functional truncated protein by skipping of the exon comprising the mutation, wherein the prophylactic or ameliorating agent contains a compound having a molecular weight of 1500 or lower. |
申请公布号 |
EP2586462(A4) |
申请公布日期 |
2013.12.11 |
申请号 |
EP20110800411 |
申请日期 |
2011.06.27 |
申请人 |
HAGIWARA, MASATOSHI;MATSUO, MASAFUMI |
发明人 |
KATAOKA, NAOYUKI;NISHIDA, ATSUSHI;HAGIWARA, MASATOSHI.;MATSUO, MASAFUMI. |
分类号 |
A61K45/00;A61K31/428;A61P21/04;A61P43/00;C07D277/64;C12N9/12;C12N9/99;C12N15/09 |
主分类号 |
A61K45/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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