Reprogramming of cholesterol sensing in epithelial cells supports pancreatic inflammation. [PDF]
Milan G +10 more
europepmc +1 more source
Miglustat in Alzheimer's Disease Associated With Heterozygous NPC1 Mutation: Exploratory Case Series and Preliminary Findings. [PDF]
Lopergolo D +10 more
europepmc +1 more source
Fighting fire with fire: PLA2G15 inhibition mobilizes BMP lipids to combat NPC1 disease. [PDF]
Meng W, Liu J, Huang X.
europepmc +1 more source
Role of mTORC1 signaling in postnatal microglia activation preceding neurodegeneration in a mouse model for Niemann-Pick disease Type C. [PDF]
Murray CE +8 more
europepmc +1 more source
Utility of 24(S)-hydroxycholesterol as a proximal biomarker to monitor long-term intrathecal adrabetadex therapy in individuals with Niemann-Pick disease, type C1. [PDF]
Porter FD +8 more
europepmc +1 more source
Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment. [PDF]
Casazza K +4 more
europepmc +1 more source
Autophagy-NAD<sup>+</sup> axis: emerging insights into neuronal homeostasis and neurodegenerative diseases. [PDF]
Kocak G +12 more
europepmc +1 more source
A Multisystem Perspective of Pediatric Cell Trafficking Disorders: Within the Cells, Beneath the Signs. [PDF]
Yoldaş Çelik M +6 more
europepmc +1 more source
Drug-Induced Reversible Lysosomal Changes Tracked in Live Cells by Holo-Tomographic Flow Cytometry. [PDF]
Pirone D +7 more
europepmc +1 more source
Niemann-Pick disease, type C1 (NPC1); NPC2 [PDF]
openaire +1 more source

