Protocol for initiation of miglustat treatment in NP-C. Reproduced
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PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

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

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

Advancing diagnosis and treatment of Niemann-Pick C disease through biomarker discovery

Small Molecules: Substrate Inhibitors, Chaperones, Stop-Codon Read Through, and Beyond - Saida Ortolano, 2016

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

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice

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

Cyclodextrins applied to the treatment of lysosomal storage disorders - ScienceDirect

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases
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