Miglustat for treatment of Niemann-Pick C disease: a randomised
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Miglustat in patients with Niemann-Pick disease Type C (NP-C): A
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology
Miglustat - Wikipedia
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
Ocular motor biomarkers in Niemann-Pick disease type C: A prospective cross-sectional multicontinental study in 72 patients
Niemann-Pick Disease Drug Type C Treatment Market Size
Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management
The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine
Niemann-Pick Disease, Type a disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials
Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
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