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Treatment of Gaucher disease with specific pharmacological chaperones and monitoring treatment using surrogate markers
Treatment of Gaucher disease with specific pharmacological chaperones and monitoring treatment using surrogate markers
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机译:用特定的药理伴侣治疗高雪氏病并使用替代标记物监测治疗
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摘要
Provided is a method of monitoring the treatment of Gaucher disease with specificpharmacological chaperones using systemic and/or cellular surrogate markers.Also provided is a new biomarker that may be used to monitor the progress of suchtreatment.
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