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

Miglustat in patients with Niemann-Pick disease Type C (NP-C): A

Miglustat - Wikipedia

Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine

Orphazyme's arimoclomol to be used with Zavesca after FDA approval

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

Niemann-Pick disease type C

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

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

IJMS, Free Full-Text

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

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

Impaired antibacterial autophagy links granulomatous intestinal inflammation in Niemann–Pick disease type C1 and XIAP deficiency with NOD2 variants in Crohn's disease

Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling - ScienceDirect

Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library

Efficacy and safety of arimoclomol in Niemann‐Pick disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatment - Mengel - 2021 - Journal of Inherited