发明公开
EP1054058A1 Pharmaceutical composition for treatment of Duchenne muscular dystrophy
审中-公开
Pharmazutische Zusammensetzung zur Behandlung von Duchenne-Muskeldystrophie
- 专利标题: Pharmaceutical composition for treatment of Duchenne muscular dystrophy
- 专利标题(中): Pharmazutische Zusammensetzung zur Behandlung von Duchenne-Muskeldystrophie
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申请号: EP00110231.8申请日: 2000-05-18
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公开(公告)号: EP1054058A1公开(公告)日: 2000-11-22
- 发明人: Matsuo, Masafumi
- 申请人: JCR PHARMACEUTICALS CO., LTD. , Matsuo, Masafumi
- 申请人地址: 3-19, Kasuga-cho Ashiya, Hyogo 659-0021 JP
- 专利权人: JCR PHARMACEUTICALS CO., LTD.,Matsuo, Masafumi
- 当前专利权人: JCR PHARMACEUTICALS CO., LTD.,Matsuo, Masafumi
- 当前专利权人地址: 3-19, Kasuga-cho Ashiya, Hyogo 659-0021 JP
- 代理机构: Reinhard - Skuhra - Weise & Partner
- 优先权: JP14093099 19990521
- 主分类号: C12N15/11
- IPC分类号: C12N15/11 ; A61K31/7125 ; A61K48/00 ; C07H21/00
摘要:
A therapeutic pharmaceutical composition for patients of Duchenne muscular dystrophy with entire loss of exon 20 in dystrophin mature mRNA is provided. The composition comprise as an active principle an antisense oligonucleotide consisting of a 20 to 50-nucleotide sequence against exon 19 of the dystrophin pre-mRNA.
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