Miglustat for treatment of Niemann-Pick C disease: a randomised
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Imaging of neuroinflammation in adult Niemann-Pick type C disease
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology
Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study, Orphanet Journal of Rare Diseases
Prevalence of dysphagia in Niemann-Pick disease type C (NP-C) and other
IJMS, Free Full-Text
Subcortical Volumetric Reductions in Adult Niemann-Pick Disease Type C: A Cross-Sectional Study
Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling - ScienceDirect
Finding pathogenic commonalities between Niemann-Pick type C and other lysosomal storage disorders: Opportunities for shared therapeutic interventions - ScienceDirect
Cyclodextrin Shows Promise in Niemann-Pick Type C Disease
Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library
Patient with Niemann-Pick disease type C: over 20 years' follow-up
New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
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
