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

Niemann- Pick Disease Type C (NPC) Market Report 2023-2033 - Wissen Research

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

5 Most Promising Companies offering hope to patients with Niemann-Pick Type C

PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

PDF] Miglustat Improves Purkinje Cell Survival and Alters Microglial Phenotype in Feline Niemann-Pick Disease Type C by Veronika M. Stein, Alexandra Crooks, Wenge Ding, Maria Prociuk, Patricia O'Donnell, Caroline Bryan, Tracey Sikora

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

Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression

Recommendations for the diagnosis and management of Niemann–Pick disease type C: An update - ScienceDirect

Dermatologic Manifestations of Niemann-Pick Disease: Background, Pathophysiology, Etiology

Niemann-Pick type C disease – the tip of the iceberg? A review of neuropsychiatric presentation, diagnosis and treatment, BJPsych Bulletin

Finding pathogenic commonalities between Niemann-Pick type C and other lysosomal storage disorders: Opportunities for shared therapeutic interventions - ScienceDirect

PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

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

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

Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management