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

Consensus clinical management guidelines for Niemann-Pick disease type C, 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

Miglustat

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

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

PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

Gastrointestinal disturbances and their management in miglustat‐treated patients - Belmatoug - 2011 - Journal of Inherited Metabolic Disease - Wiley Online Library

Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrin. - Abstract - Europe PMC

Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1 – topic of research paper in Clinical medicine. Download scholarly article

Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02)

SEC Filing Orphazyme A/S