Critical role for glycosphingolipids in Niemann-Pick disease type
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PDF] Niemann-Pick Type C Disease—Pathophysiology and Future Perspectives for Treatment
Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine
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Miglustat Reverts the Impairment of Synaptic Plasticity in a Mouse Model of NPC Disease
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Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling - ScienceDirect
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Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016