Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith

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专利权人:

PTC THERAPEUTICS; INC.

发明人:

ALMSTEAD, NEIL G

申请号：

NZ57189907

公开号：

NZ571899A

申请日:

2007.03.29

申请国别(地区):

NZ

年份:

2012

代理人:

摘要：

Provided is the use of the compound of formula (I) as depicted, with defined substituents, in the preparation of a medicament for use in a subject for the treatment of duchenne muscular dystrophy, wherein the subject has a nonsense mutation in the codon corresponding to position 1417, 3625, or 492 of the dystrophin protein.

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中国工程科技知识中心

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http://www.ckcest.cn/home/