The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency.
Archives of medical science : AMS(2023)
摘要
Future research with a larger MSD population and a longer-term follow-up is warranted to determine whether trehalose can improve MSD patient health and clinical outcomes.
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关键词
lysosomal storage diseases, multiple sulfatase deficiency, trehalose
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