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The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency
oleh: Shabnam Radbakhsh, Moein Mobini, Eric Gumpricht, Maciej Banach, Tannaz Jamialahmadi, Amirhossein Sahebkar
Format: | Article |
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Diterbitkan: | Termedia Publishing House 2023-01-01 |
Deskripsi
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. Material and 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.