Critical role for glycosphingolipids in Niemann-Pick disease type
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Niemann–Pick disease, type C - Wikipedia
mRNA Treatment Rescues Niemann-Pick Disease Type C1 in Patient Fibroblasts
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Sphingolipid - Wikipedia
mRNA Treatment Rescues Niemann-Pick Disease Type C1 in Patient Fibroblasts
Development of a bile acid–based newborn screen for Niemann-Pick disease type C
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Critical role for glycosphingolipids in Niemann-Pick disease type C: Current Biology
IJMS, Free Full-Text
PDF] Niemann-Pick Type C Disease—Pathophysiology and Future Perspectives for Treatment
Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016
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