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

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

Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology

Advancing diagnosis and treatment of Niemann-Pick C disease through biomarker discovery

IJMS, Free Full-Text

Drug-induced increase in lysobisphosphatidic acid reduces the cholesterol overload in Niemann-Pick type C cells and mice

Loss of NPC1 enhances phagocytic uptake and impairs lipid trafficking in microglia

Full article: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat

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

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