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

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

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

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

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

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

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

IJMS, Free Full-Text

Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016

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

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

Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult

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

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