Protocol for initiation of miglustat treatment in NP-C. Reproduced
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Pluronic based β-cyclodextrin polyrotaxanes for treatment of Niemann-Pick Type C disease
Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult
Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases
Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice
Necroptosis in Niemann–Pick disease, type C1: a potential therapeutic target
Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases
Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late- Onset Pompe Disease
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