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

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

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

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

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

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

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

Unbiased yeast screens identify cellular pathways affected in Niemann–Pick disease type C

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

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

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice

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

Prevalence of dysphagia in Niemann-Pick disease type C (NP-C) and other

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