Therapeutic intervention for Niemann–Pick type C disease must target
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Progression of Niemann–Pick type C disease in mice and humans
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Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management
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
IJMS, Free Full-Text
Clinical, ocular motor, and imaging profile of Niemann-Pick type C heterozygosity
Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
Combination therapy a potential strategy for treating Niemann-Pick disease
Severely impaired CTL killing is a feature of the neurological disorder Niemann-Pick disease type C1 - ScienceDirect
Niemann-Pick disease type C, Radiology Reference Article
PDF) Recent Advances in the Diagnosis and Treatment of Niemann-Pick Disease Type C in Children: A Guide to Early Diagnosis for the General Pediatrician
Proposed pathological cascade in Niemann-Pick disease type C (NPC)
Experimental treatment for Niemann-Pick disease type C1 appears safe, effective
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