申请人:Children's Hospital Medical Center
公开号:US10751387B2
公开(公告)日:2020-08-25
The instant disclosure relates to methods and compositions for the treatment of Gaucher disease, particularly type II and III neuronopathic Gaucher disease (nGD). The methods include the step of administering to an individual in need thereof an effective amount of a ryanodine receptor inhibitor or a pharmaceutically acceptable salt thereof.
本公开涉及治疗戈谢病,特别是 II 型和 III 型神经病性戈谢病(nGD)的方法和组合物。这些方法包括向有需要的个体施用有效量的雷诺丁受体抑制剂或其药学上可接受的盐。