Therapeutic intervention for Niemann–Pick type C disease must target

Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine

The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine

mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts

Main therapies and point of action in Niemann type C disease.

Niemann-Pick disease type C, Radiology Reference Article

Recommendations on the diagnosis and management of Niemann-Pick disease type C - ScienceDirect

Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine

Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice