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- Title
The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency.
- Authors
Radbakhsh, Shabnam; Mobini, Moein; Gumpricht, Eric; Banach, Maciej; Jamialahmadi, Tannaz; Sahebkar, Amirhossein
- Abstract
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.
- Subjects
SULFATASES; INTRAVENOUS therapy; TREHALOSE; LYSOSOMAL storage diseases
- Publication
Archives of Medical Science, 2023, Vol 19, Issue 5, p1564
- ISSN
1734-1922
- Publication type
Case Study
- DOI
10.5114/aoms/159711