Miglustat for treatment of Niemann-Pick C disease: a randomised
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Miglustat, C10H21NO4
Miglustat in patients with Niemann-Pick disease Type C (NP-C): A
Niemann–Pick disease - Wikipedia
Full article: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
5 Most Promising Companies offering hope to patients with Niemann-Pick Type C
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
Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult
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
Recommendations for the diagnosis and management of Niemann–Pick disease type C: An update - ScienceDirect
Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases
Rapid Diagnosis of 83 Patients with Niemann Pick Type C Disease and Related Cholesterol Transport Disorders by Cholestantriol Screening - eBioMedicine
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