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

Miglustat

Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

IJMS, Free Full-Text

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

Recommendations for oral treatment for adult patients with type 1 Gaucher disease

Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression

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

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

IJMS, Free Full-Text

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

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

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