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Pharmacological therapy for neuronopathic Gaucher disease
Pharmacological therapy for neuronopathic Gaucher disease
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机译:神经性戈谢病的药物治疗
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摘要
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.
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