Miglustat for treatment of Niemann-Pick C disease: a randomised
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Influence of miglustat therapy on NP-C patient survival. Kaplan-Meier
Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016
Prevalence of dysphagia in Niemann-Pick disease type C (NP-C) and other
The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine
Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
Full article: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
IJMS, Free Full-Text
Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrin. - Abstract - Europe PMC
Miglustat - Wikipedia
PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study
Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult
Orphazyme's arimoclomol to be used with Zavesca after FDA approval
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