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

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

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

Histone deacetylase inhibitor treatment dramatically reduces cholesterol accumulation in Niemann-Pick type C1 mutant human fibroblasts

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

Effects of Miglustat on Stabilization of Neurological Disorder in Niemann–Pick Disease Type C: Iranian Pediatric Case Series - Parvaneh Karimzadeh, Seyed Hassan Tonekaboni, Mahmoud Reza Ashrafi, Yousef Shafeghati, Alireza Rezayi, Shadab Salehpour

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

Niemann–Pick Disease Type C: Induced Pluripotent Stem Cell–Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy - Daozhan Yu, Manju Swaroop, Mengqiao Wang, Ulrich Baxa, Rongze Yang, Yiping Yan, Turhan

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Niemann- Pick Disease Type C (NPC) Market Report 2023-2033 - Wissen Research

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

Niemann-Pick Disease disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials

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