Benny Liu
Abstract
mice, neurodegeneration was completely prevented. This review will explore the pathophysiology of NPC disease and the use of 2HPBCD as a possible therapeutic modality.
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mice, neurodegeneration was completely prevented. This review will explore the pathophysiology of NPC disease and the use of 2HPBCD as a possible therapeutic modality.
Key concepts: NPC1, Neurodegeneration, Lysosomal storage disease, Niemann–Pick disease, type C, Niemann–Pick disease, Endosome, Disease, Lysosome