Protocol for initiation of miglustat treatment in NP-C. Reproduced

PDF) Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C

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A novel strategy for delivering Niemann‐Pick type C2 proteins across the blood–brain barrier using the brain endothelial‐specific AAV‐BR1 virus - Rasmussen - 2023 - Journal of Neurochemistry - Wiley Online Library

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