Miglustat for treatment of Niemann-Pick C disease: a randomised
Por um escritor misterioso
Last updated 23 fevereiro 2025
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Niemann- Pick Disease Type C (NPC) Market Report 2023-2033 - Wissen Research
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Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study, Orphanet Journal of Rare Diseases
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5 Most Promising Companies offering hope to patients with Niemann-Pick Type C
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PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study
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PDF] Miglustat Improves Purkinje Cell Survival and Alters Microglial Phenotype in Feline Niemann-Pick Disease Type C by Veronika M. Stein, Alexandra Crooks, Wenge Ding, Maria Prociuk, Patricia O'Donnell, Caroline Bryan, Tracey Sikora
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Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology
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Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
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Recommendations for the diagnosis and management of Niemann–Pick disease type C: An update - ScienceDirect
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Dermatologic Manifestations of Niemann-Pick Disease: Background, Pathophysiology, Etiology
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Niemann-Pick type C disease – the tip of the iceberg? A review of neuropsychiatric presentation, diagnosis and treatment, BJPsych Bulletin
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Finding pathogenic commonalities between Niemann-Pick type C and other lysosomal storage disorders: Opportunities for shared therapeutic interventions - ScienceDirect
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PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study
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Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
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New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
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Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management
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