CLINICAL PHARMACOLOGY / RESEARCH LETTER
The Effect of Intravenous Trehalose Administration in a Patient with Multiple Sulfatase Deficiency: A Case Study
| 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 | Department of Preventive Cardiology and Lipidology, Medical University of Lodz (MUL), Lodz, Poland |
| 5 | Cardiovascular Research Centre, University of Zielona Gora, Zielona Gora, Poland. |
| 6 | Department of Nutrition, Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran |
| 7 | Biotechnology Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran |
CORRESPONDING AUTHOR
Submission date: 2022-12-26
Acceptance date: 2023-01-23
Online publication date: 2023-01-23
KEYWORDS
TOPICS
ABSTRACT
Introduction:
We describe the case of a female child with MSD who received intravenous (IV) trehalose (15 g/week) for three months.
Material and 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 three months intervention.
Conclusions:
Future research with a larger MSD population and a longer-term follow-up is warranted to determine the if trehalose can improve MSD patient health and clinical outcomes.
We describe the case of a female child with MSD who received intravenous (IV) trehalose (15 g/week) for three months.
Material and 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 three months intervention.
Conclusions:
Future research with a larger MSD population and a longer-term follow-up is warranted to determine the if trehalose can improve MSD patient health and clinical outcomes.
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