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

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Recent advances in the treatment of Niemann pick disease type C: A mini-review - ScienceDirect

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

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

Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling - 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

Influence of miglustat therapy on NP-C patient survival. Kaplan-Meier

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

The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine