| 发明名称 |
METHODS FOR TREATING SPINAL MUSCULAR ATROPHY |
| 摘要 |
Described herein are methods for the identification or validation of compounds capable of causing ribosomal frameshifting and the use of said compounds to produce a stabilized SMNΔEx7 protein and treat Spinal Muscular Dystrophy. |
| 申请公布号 |
US2015283115(A1) |
申请公布日期 |
2015.10.08 |
| 申请号 |
US201514621051 |
申请日期 |
2015.02.12 |
| 申请人 |
PTC THERAPEUTICS, INC. |
发明人 |
Paushkin Sergey V.;Naryshkin Nikolai A.;Welch Ellen |
| 分类号 |
A61K31/4035 |
主分类号 |
A61K31/4035 |
| 代理机构 |
|
代理人 |
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| 主权项 |
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| 地址 |
South Plainfield NJ US |
