Protocol for initiation of miglustat treatment in NP-C. Reproduced

Dysphagia as a risk factor for mortality in Niemann-Pick disease type C: systematic literature review and evidence from studies with miglustat, Orphanet Journal of Rare Diseases

Miglustat

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Protocol for initiation of miglustat treatment in NP-C. Reproduced with

A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1, Lipids in Health and Disease

Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders - ScienceDirect

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

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