Introduction: We describe the case of a female child with multiple sulfatase deficiency (MSD) who received intravenous (IV) trehalose (15 g/week) for 3 months.
Methods: The efficacy of trehalose was evaluated by comparing serum biomarkers, a quality-of-life questionnaire (HRQoL), and imaging (brain MRI and ultrasonography of liver and spleen) at weeks 0 (W0) and 12 (W12).
Results: The improvement in quality of life assessments along with a slight decrease in the spleen dimensions were observed after 3-month intervention.
Conclusions: 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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| http://dx.doi.org/10.5114/aoms/159711 | DOI Listing |
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