(EN) The present description relates'to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.
(ZH) 本说明书涉及用于改善细胞中的前体‑mRNA剪接的式(I)化合物。特别地,本说明书的另一方面涉及取代噻吩并[3,2‑b]吡啶化合物、其形式和药物组合物以及用于治疗或改善家族性自主神经功能障碍的方法。