Disclosed are compounds of Formula I Formula (I) in which n and R1?-R4? 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.
本文披露了公式I的化合物(I),其中n和R1-R4如本文所定义。还披露了一种治疗脊髓肌肉萎缩症的方法,以及使用这些化合物来增加SMN表达、增加E
AAT2表达或增加直接或间接由突变或移码引入的翻译终止密码子编码的核酸表达的方法。