Therapeutic intervention for Niemann–Pick type C disease must target

Juvenile CLN3 disease is a lysosomal cholesterol storage disorder: similarities with Niemann-Pick type C disease - eBioMedicine

Niemann-Pick type C disease: molecular mechanisms and potential therapeutic approaches. - Abstract - Europe PMC

Niemann-Pick disease A or B in four pediatric patients and SMPD1 mutation carrier frequency in the Mexican population

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

Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

IJMS, Free Full-Text

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

Adult onset Niemann-Pick disease type C presenting with psychosis

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