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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 |
Publicado: |
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 |