https://www.selleckchem.com/pr....oducts/BafilomycinA1
Niemann-Pick type C (NP-C) disease is a neurovisceral atypical lysosomal lipid storage disorder with a poor prognosis. We present the 5-year neuropsychological follow-up of a patient with juvenile onset NP-C, spanning the pre-diagnostic stage to the period after treatment with miglustat (Actelion Pharmaceuticals Inc., CA, US). In the initial stages of the disease, the patient presented behavioral dy****ecutive symptoms resembling those frequently observed in adult-onset forms and frontotemporal dementia, which frequently makes earl