Abstract:Niemann-Pick (NP) types A (NPA) and B (NPB) are life-threatening and rare inherited lysosomal storage disorders, caused by a deficiency in the acid sphingomyelinase enzyme activity. The negative outcome of this deficiency is the sphingomyelin (SM) accumulation in different organs and tissues. Trehalose is a natural disaccharide with neuroprotective and autophagy-inducing abilities which has recently been shown to improve clinical and biochemical features of patients with NPA/B. Changes in the serum metabolome … Show more
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