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Updated: Jul 16, 2025

Rapid One-step Enzymatic Synthesis and All-aqueous Purification of Trehalose Analogues
Published on: February 17, 2017
The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency
Shabnam Radbakhsh1, Moein Mobini2, Eric Gumpricht3
1Student Research Committee, Mashhad University of Medical Sciences, Mashhad, Iran.
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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