Miglustat for treatment of Niemann-Pick C disease: a randomised
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Miglustat - Wikipedia
A first therapy for Niemann-Pick C - The Lancet Neurology
Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrin. - Abstract - Europe PMC
PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study
Recent advances in the treatment of Niemann pick disease type C: A mini-review - ScienceDirect
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
Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
Niemann-Pick type C disease – the tip of the iceberg? A review of neuropsychiatric presentation, diagnosis and treatment, BJPsych Bulletin
PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study
Orphazyme's arimoclomol to be used with Zavesca after FDA approval
Cost-effectiveness of miglustat versus symptomatic therapy of Niemann–Pick disease type C
Necroptosis in Niemann–Pick disease, type C1: a potential therapeutic target
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