Miglustat for treatment of Niemann-Pick C disease: a randomised

Imaging of neuroinflammation in adult Niemann-Pick type C disease

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

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

Cureus, Niemann-Pick Disease: An Approach for Diagnosis in Adulthood

Rapid Diagnosis of 83 Patients with Niemann Pick Type C Disease and Related Cholesterol Transport Disorders by Cholestantriol Screening - eBioMedicine

Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study, Orphanet Journal of Rare Diseases

The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine

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

Pharmaceutical Benefits Scheme (PBS) Miglustat, capsule, 100 mg, Zavesca® - November 2011

Cost-effectiveness of miglustat versus symptomatic therapy of Niemann–Pick disease type C

Miglustat, C10H21NO4

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