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

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice

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

Phenotypic expression of swallowing function in Niemann–Pick disease type C1, Orphanet Journal of Rare Diseases

A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1, Lipids in Health and Disease

Elucidating the mechanism of cyclodextrins in the treatment of Niemann-Pick Disease Type C using crosslinked 2-hydroxypropyl-β-cyclodextrin

Gastrointestinal disturbances and their management in miglustat-treated patients

Miglustat

NPC1 enables cholesterol mobilization during long‐term potentiation that can be restored in Niemann–Pick disease type C by CYP46A1 activation

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage 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

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

PDF) Goal-oriented therapy with miglustat in Gaucher disease