NIEMANN PICK C

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

Niemann-Pick disease Type C - causes, symptoms, diagnosis, treatment, pathology

Niemann-Pick Type C Disease Reveals a Link between Lysosomal Cholesterol and PtdIns(4,5)P2 That Regulates Neuronal Excitability - ScienceDirect

Estimated prevalence of Niemann–Pick type C disease in Quebec

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology

The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine

Lipid trafficking defects in Niemann-Pick type C disease

Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult

Infantile form of Niemann-Pick disease type C with demyelination: An uncommon feature

Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium

Critical role for glycosphingolipids in Niemann-Pick disease type C: Current Biology

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

Niemann-Pick disease type C

The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine

Niemann-Pick disease type C, Orphanet Journal of Rare Diseases