Protocol for initiation of miglustat treatment in NP-C. Reproduced
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Protocol for initiation of miglustat treatment in NP-C. Reproduced with
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult
Acat1/Soat1 knockout extends the mutant Npc1 mouse lifespan and ameliorates functional deficiencies in multiple organelles of mutant cells
Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases
Substrate reduction therapy in the infantile form of Tay-Sachs disease
IJMS, Free Full-Text
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
Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice
Cyclodextrins applied to the treatment of lysosomal storage disorders - ScienceDirect
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology
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