Disclosed are new small molecules having a 4-methylpyrrrolo[l,2-a]pyrimidine- 8-carboxamide core structure and the uses thereof for modulating glucocerebrosidase activity. Also disclosed are pharmaceutical compositions comprising the small molecules which may be administered in methods of treating diseases or disorders associated with glucocerebrosidase activity, including neurological diseases and disorders such as Gaucher's disease and Parkinson's disease. The small molecules may contain a fluorophore or may be conjugated to a fluorophore in order to prepare a fluorescent probe for use in high throughput screening methods to identify new modulators of glucocerebrosidase activity via fluorescence polarization.
揭示了具有4-甲基
吡咯并[1,2-a]
嘧啶-8-羧酰胺核结构的新型小分子及其用途,用于调节
葡萄糖乙酰
硫脂酶活性。还揭示了包含这些小分子的药物组合物,可用于治疗与
葡萄糖乙酰
硫脂酶活性相关的疾病或疾病,包括神经系统疾病和障碍,如高雪氏病和帕
金森病。这些小分子可能含有荧光团或与荧光团结合,以制备用于高通量筛选方法中的荧光探针,以通过荧光极化识别
葡萄糖乙酰
硫脂酶活性的新调节剂。