Miglustat for treatment of Niemann-Pick C disease: a randomised
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The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine
Pharmaceutical Benefits Scheme (PBS) Miglustat, capsule, 100 mg, Zavesca® - November 2011
Dermatologic Manifestations of Niemann-Pick Disease: Background, Pathophysiology, Etiology
Ocular motor biomarkers in Niemann-Pick disease type C: A prospective cross-sectional multicontinental study in 72 patients
Subcortical Volumetric Reductions in Adult Niemann-Pick Disease Type C: A Cross-Sectional Study
Cost-effectiveness of miglustat versus symptomatic therapy of Niemann–Pick disease type C
Recommendations for the diagnosis and management of Niemann–Pick disease type C: An update - ScienceDirect
Unbiased yeast screens identify cellular pathways affected in Niemann–Pick disease type C
IJMS, Free Full-Text
Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine
Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management
Imaging of neuroinflammation in adult Niemann-Pick type C disease
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