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新型小分子向SCN1A的开发与婴儿期严重肌痛性相关

Dong Gun Kim1, Kyu-Seok Hwang2, Se Hwan Ahn1

  • 1Department of Chemistry, Gwangju Institute of Science and Technology, Gwangju 61005, Republic of Korea.

Journal of medicinal chemistry
|January 23, 2026
PubMed
概括

一种新型化合物20e在斑马鱼和小鼠模型中有效地减少了婴儿严重肌细胞性 (SMEI) 的发作. 这种有前途的候选药物还使神经元活动正常化,并显示出良好的安全性.

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