Main therapies and point of action in Niemann type C disease.
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Impaired Autophagy in the Lipid-Storage Disorder Niemann-Pick Type

Miglustat for treatment of Niemann-Pick C disease: a randomised

PDF] Niemann-Pick Type C Disease—Pathophysiology and Future

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Molecular mechanism(s) of neurodegeneration in Niemann-Pick type C

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Niemann-Pick Disease Type C - cyclotherapeutics
mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient

Critical role for glycosphingolipids in Niemann-Pick disease type
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