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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Detalles Bibliográficos
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:
Research Letter
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
Descripción
Sumario: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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