Therapeutic intervention for Niemann–Pick type C disease must target

Finding pathogenic commonalities between Niemann-Pick type C and other lysosomal storage disorders: Opportunities for shared therapeutic interventions - ScienceDirect

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

Niemann-Pick Disease Type C (NP-C) Gene Therapy Activation Meeting (GAM) - International Niemann-Pick Disease Alliance (INPDA)

The Neuropsychiatry of Niemann-Pick Type C Disease in Adulthood

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial - The Lancet

Clinical, ocular motor, and imaging profile of Niemann-Pick type C heterozygosity

Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine

Niemann–Pick type C disease as proof‐of‐concept for intelligent biomarker panel selection in neurometabolic disorders - Papandreou - 2022 - Developmental Medicine & Child Neurology - Wiley Online Library

IJMS, Free Full-Text

Niemann-Pick disease type C – topic of research paper in Biological sciences. Download scholarly article PDF and read for free on CyberLeninka open science hub.

Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases

Niemann-Pick disease type C, Radiology Reference Article

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

Recommendations for the detection and diagnosis of Niemann-Pick disease type C

Adult onset Niemann-Pick disease type C presenting with psychosis