Therapeutic intervention for Niemann–Pick type C disease must target
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Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library
mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts
Identification of Novel Biomarkers for Niemann–Pick Disease Using Gene Expression Analysis of Acid Sphingomyelinase Knockout Mice: Molecular Therapy
Niemann-Pick Disease Type C Gene Therapy Gets Rare Pediatric Disease Designation
Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
Niemann-Pick disease type C as a neurovisceral disease. Schematic
Lysosomal and Mitochondrial Liaisons in Niemann-Pick Disease
Adult‐onset Niemann–Pick disease type C masquerading as spinocerebellar ataxia - Vo - 2022 - Molecular Genetics & Genomic Medicine - Wiley Online Library
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
IJMS, Free Full-Text
Frontiers c-Abl Activation Linked to Autophagy-Lysosomal Dysfunction Contributes to Neurological Impairment in Niemann-Pick Type A Disease
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