Mutations in NPC1 cause Niemann-Pick Type C. Mice lacking NPC1 only in microglia have rampant neuroinflammation ... such as neuronal loss in the cerebellum and demyelination at late stages of the ...

Exosomes, nano-sized extracellular vesicles secreted by all cells, are a fundamental part of the human body’s internal ... development for Niemann–Pick disease type C, a disorder in which ...

Berry-Kravis was able to make a request to the FDA so that Charlie could become the first human in the world ... awareness of any rare disease,” Harry said. Niemann-Pick Disease is a rare ...