Main therapies and point of action in Niemann type C disease.
Descrição
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Understanding the phenotypic variability in Niemann-Pick disease
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PDF] Niemann-Pick Type C Disease—Pathophysiology and Future
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Niemann-Pick disease type C1 is a sphingosine storage disease that
Acid Sphingomyelinase Deficiency - Symptoms, Causes, Treatment
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Psychiatric and Cognitive Symptoms Associated with Niemann-Pick
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Immune dysfunction in Niemann‐Pick disease type C - Platt - 2016
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Niemann–Pick Disease Type C: Induced Pluripotent Stem Cell–Derived
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Niemann-Pick disease type C Orphanet Journal of Rare Diseases
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Miglustat in Niemann-Pick disease type C patients: a review
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Niemann–Pick type C disease as proof‐of‐concept for intelligent
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5 Most Promising Companies offering hope to patients with Niemann
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Correlation of age of onset and clinical severity in Niemann–Pick
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Combination therapy a potential strategy for treating Niemann-Pick
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Niemann-Pick Type C Disease - Pipeline Insight, 2021
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