Rao Fu

TL;DR: Blood concentrations of two related oxysterols molecules were almost 10 times higher in Niemann-Pick C1 patients than in age-matched healthy controls or those with other diseases such as atherosclerosis or diabetes, suggesting that the two oxysterol molecules are accurate diagnostic markers of early clinical disease and can be used not only to monitor disease progression but also to demonstrate drug efficacy.

TL;DR: Evaluation of serum from 37 NPC patients showed significant decreases in both the fraction of reduced coenzyme Q10 (CoQ10) and trolox equivalent antioxidant capacity (TEAC) and both findings are consistent with increased oxidative stress in NPC.

TL;DR: Two secreted proteins, galectin-3 (LGALS3), a pro-inflammatory molecule, and cathepsin D (CTSD), a lysosomal aspartic protease, have the potential to aid in diagnosis and serve as biomarkers to monitor efficacy in therapeutic trials.

TL;DR: The mouse model data suggest that the distinct antioxidant effects of NAC could provide potential treatment of NPC1 disease, possibly in concert with other therapeutic molecules at earlier stages of disease progression, and validated the NPC1ASO mouse as an efficient model for candidate NPC1 drug screening.

TL;DR: It is found that resveratrol repaired the fission–fusion balance, autophagic flux and mitochondrial biosynthesis compared by H/R group, followed by increased functional mitochondrial number, mitochondrial bioenergetics and a better mitochondrial antioxidant enzyme system.