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

Miglustat - Wikipedia

mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts

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

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

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology

Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

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

Subcortical Volumetric Reductions in Adult Niemann-Pick Disease Type C: A Cross-Sectional Study

New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat

Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry, Orphanet Journal of Rare Diseases

New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat

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

Unbiased yeast screens identify cellular pathways affected in Niemann–Pick disease type C