CLINICAL PHARMACOLOGY / RESEARCH LETTER
 
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.

eISSN:1896-9151
ISSN:1734-1922