Niemann-Pick Disease Type C in Adulthood – A Psychiatric and Neurological Disorder
require the introduction of more human data on illness effects and progression to merge with the existing animal models. Expansion of non-invasive neuroimaging techniques will be needed to allow for the probing of brain changes in vivo.31,64
Currently, miglustat remains
the only disease-specific drug available for adults and children suffering from NPC. The high rate of schizophrenia-like psychosis in NPC also makes it an interesting disease model and illness phenocopy to study with the aim of further understanding the neuropathology of major mental disorders. n
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