This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of small-molecules having a phenyl-sulfonic amide (or similar) structure which function as agonists of mucolipin 1 (ML1), and their use as therapeutics for the treatment of Duchenne muscular dystrophy (DMD) and related disorders.
这项发明属于药物
化学领域。特别是,该发明涉及一类新的小分子,具有
苯磺
酰胺(或类似)结构,作为肌球脂蛋白1(ML1)的激动剂,以及它们作为治疗杜氏肌肉萎缩症(
DMD)和相关疾病的疗法的用途。