Disclosed is a compound of Formula (I) in which W and R1-R6 are defined herein. Also disclosed is a method of treating spinal muscular atrophy, as well as methods of using such compounds to increase SMN expression, increase EAAT2 expression, or increase the expression of a nucleic acid that encodes a translational stop codon introduced directly or indirectly by mutation or frameshift.
公开的是一种化合物,其
化学式为(I),其中W和R1-R6在此处被定义。还公开了一种治疗脊髓性肌萎缩症的方法,以及使用这种化合物增加SMN表达、增加E
AAT2表达,或增加通过突变或移码直接或间接引入的编码翻译终止密码子的核酸表达的方法。