Therapeutic intervention for Niemann–Pick type C disease must target
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Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
Experimental treatment for Niemann-Pick disease type C1 appears safe, effective
Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
Niemann-Pick type C disease: molecular mechanisms and potential therapeutic approaches. - Abstract - Europe PMC
Main therapies and point of action in Niemann type C disease.
IJMS, Free Full-Text
Frontiers Case Report: Be Aware of “New” Features of Niemann–Pick Disease: Insights From Two Pediatric Cases
Niemann-Pick Disease, Type B disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials
IJMS, Free Full-Text
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