The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency-Reference-Cited by-同舟云学术

The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency

Published:2023-01-23 Issue:5 Volume:19 Page:1564-1568
ISSN:1734-1922
Container-title:Archives of Medical Science
language:
Short-container-title:Arch Med Sci

Author:

Radbakhsh Shabnam¹^ORCID,Mobini Moein²^ORCID,Gumpricht Eric³^ORCID,Banach Maciej⁴^ORCID,Jamialahmadi Tannaz⁵,Sahebkar Amirhossein⁵⁶^ORCID

Affiliation:

1. Student Research Committee, Mashhad University of Medical Sciences, Mashhad, Iran

2. Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran

3. Isagenix International LLC, Gilbert, AZ, United States

4. Cardiovascular Research Centre, University of Zielona Gora, Zielona Gora, Poland

5. Applied Biomedical Research Center, Mashhad University of Medical Sciences, Mashhad, Iran

6. Biotechnology Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran

Abstract

IntroductionWe 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 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.

Publisher

Termedia Sp. z.o.o.

Subject

General Medicine

Link

https://www.archivesofmedicalscience.com/pdf-159711-94249

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1. Effect of trehalose on mortality and disease severity in ICU-admitted patients: Protocol for a triple-blind, randomized, placebo-controlled clinical trial;Contemporary Clinical Trials Communications;2024-08

2. Anti-arthritic and Antioxidant Effects of Trehalose in an Experimental Model of Arthritis;Recent Advances in Inflammation & Allergy Drug Discovery;2023-09