发明名称 USE OF NONSENSE CODON SUPPRESSOR COMPOUNDS FOR THE PREPARATION OF A MEDICAMENT FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY OR CYSTIC FIBROSIS
摘要 <p>The present invention relates to functional proteins encoded by nucleic acid sequences comprising a nonsense mutation. The present invention also relates to methods for the production of functional proteins encoded by nucleic acid sequences comprising a nonsense mutation and the use of such proteins for prevention, management and/or treatment of diseases associated with a nonsense mutation(s) in a gene.</p>
申请公布号 IL222557(D0) 申请公布日期 2012.12.31
申请号 IL20120222557 申请日期 2012.10.18
申请人 PTC THERAPEUTICS, INC 发明人
分类号 A61K 主分类号 A61K
代理机构 代理人
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