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- Shabnam Radbakhsh, Moein Mobini, Eric Gumpricht, Maciej Banach, Tannaz Jamialahmadi, and Amirhossein Sahebkar.
- Student Research Committee, Mashhad University of Medical Sciences, Mashhad, Iran.
- Arch Med Sci. 2023 Jan 1; 19 (5): 156415681564-1568.
IntroductionWe describe the case of a female child with multiple sulfatase deficiency (MSD) who received intravenous (IV) trehalose (15 g/week) for 3 months.MethodsThe 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).ResultsThe improvement in quality of life assessments along with a slight decrease in the spleen dimensions were observed after 3-month intervention.ConclusionsFuture 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.Copyright: © 2023 Termedia & Banach.
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