Protocol for initiation of miglustat treatment in NP-C. Reproduced
Por um escritor misterioso
Last updated 05 julho 2024
PDF) Goal-oriented therapy with miglustat in Gaucher disease
Loss of NPC1 enhances phagocytic uptake and impairs lipid trafficking in microglia
Oxidative stress: a pathogenic mechanism for Niemann-Pick type C disease. - Abstract - Europe PMC
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
SEC Filing Orphazyme A/S
NPC1 enables cholesterol mobilization during long‐term potentiation that can be restored in Niemann–Pick disease type C by CYP46A1 activation
Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study, Orphanet Journal of Rare Diseases
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology
Drug-induced increase in lysobisphosphatidic acid reduces the cholesterol overload in Niemann-Pick type C cells and mice
Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases
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