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

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...

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Autores principales: Radbakhsh, Shabnam, Mobini, Moein, Gumpricht, Eric, Banach, Maciej, Jamialahmadi, Tannaz, Sahebkar, Amirhossein
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Termedia Publishing House 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10507772/
https://www.ncbi.nlm.nih.gov/pubmed/37732042
http://dx.doi.org/10.5114/aoms/159711
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author Radbakhsh, Shabnam
Mobini, Moein
Gumpricht, Eric
Banach, Maciej
Jamialahmadi, Tannaz
Sahebkar, Amirhossein
author_facet Radbakhsh, Shabnam
Mobini, Moein
Gumpricht, Eric
Banach, Maciej
Jamialahmadi, Tannaz
Sahebkar, Amirhossein
author_sort Radbakhsh, Shabnam
collection PubMed
description 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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spelling pubmed-105077722023-09-20 The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency Radbakhsh, Shabnam Mobini, Moein Gumpricht, Eric Banach, Maciej Jamialahmadi, Tannaz Sahebkar, Amirhossein Arch Med Sci Research Letter 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. Termedia Publishing House 2023-01-23 /pmc/articles/PMC10507772/ /pubmed/37732042 http://dx.doi.org/10.5114/aoms/159711 Text en Copyright: © 2023 Termedia & Banach https://creativecommons.org/licenses/by-nc-sa/4.0/This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-ShareAlike 4.0 International (CC BY-NC-SA 4.0) License, allowing third parties to copy and redistribute the material in any medium or format and to remix, transform, and build upon the material, provided the original work is properly cited and states its license.
spellingShingle Research Letter
Radbakhsh, Shabnam
Mobini, Moein
Gumpricht, Eric
Banach, Maciej
Jamialahmadi, Tannaz
Sahebkar, Amirhossein
The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency
title The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency
title_full The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency
title_fullStr The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency
title_full_unstemmed The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency
title_short The effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency
title_sort effect of intravenous trehalose administration in a patient with multiple sulfatase deficiency
topic Research Letter
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10507772/
https://www.ncbi.nlm.nih.gov/pubmed/37732042
http://dx.doi.org/10.5114/aoms/159711
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