Therapeutic intervention for Niemann–Pick type C disease must target
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Niemann-Pick Disease Type C Gene Therapy Gets Rare Pediatric Disease Designation
Niemann–Pick Disease Type C: Induced Pluripotent Stem Cell–Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy - Daozhan Yu, Manju Swaroop, Mengqiao Wang, Ulrich Baxa, Rongze Yang, Yiping Yan, Turhan
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
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
Proposed pathological cascade in Niemann-Pick disease type C (NPC)
Niemann-Pick type C disease: molecular mechanisms and potential therapeutic approaches. - Abstract - Europe PMC
5 Most Promising Companies offering hope to patients with Niemann-Pick Type C
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Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
IJMS, Free Full-Text
Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann–Pick Type C Using Quantitative Proteomics
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