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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...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Termedia Publishing House
2023
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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. |
format | Online Article Text |
id | pubmed-10507772 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Termedia Publishing House |
record_format | MEDLINE/PubMed |
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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