Long-term effects of eliglustat on skeletal manifestations in clinical trials of patients with Gaucher disease type 1.

Genetics in medicine : official journal of the American College of Medical Genetics（2023）

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摘要

These findings confirm the long-term efficacy of eliglustat on skeletal complications of Gaucher disease in treatment-naïve and ERT-switch patients.

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关键词

Acid β-glucosidase deficiency,Eliglustat,Gaucher disease type 1,Skeletal manifestations,Substrate reduction therapy

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