Miglustat for treatment of Niemann-Pick C disease: a randomised
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Full article: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
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
The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine
Niemann-Pick disease type C clinical database: cognitive and coordination deficits are early disease indicators – topic of research paper in Clinical medicine. Download scholarly article PDF and read for free on CyberLeninka
Unbiased yeast screens identify cellular pathways affected in Niemann–Pick disease type C
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology
IJMS, Free Full-Text
Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance of Cholesterol Accumulation in Niemann-Pick C1 Fibroblasts
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology
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
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