Impact of Intravenous Trehalose Administration in Patients with Niemann-Pick Disease Types A and B.

Moein MobiniShabnam RadbakhshFrancyne KubaskiPeyman EshraghiSaba VakiliRahim VakiliManijeh KhaliliMajid VaresvazirianTannaz JamialahmadiSeyed Ali AlamdaranSeyed Javad SayediOmid RajabiSeyed Ahmad EmamiŽeljko ReinerAmir Hossein Sahebkar

Published in: Journal of clinical medicine (2022)

Positive trends in health-related quality of life (HRQoL), serum biomarkers, and organomegaly were observed after 3 months of treatment with trehalose in patients with NPA and NPB. Although not statistically significant, due to the small number of patients enrolled, these results are encouraging and should be further explored.

Keyphrases

end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
peritoneal dialysis
prognostic factors
high dose
low dose
patient reported outcomes
replacement therapy