Miglustat for treatment of Niemann-Pick C disease: a randomised

Main therapies and point of action in Niemann type C disease.

Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling - ScienceDirect

Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry, Orphanet Journal of Rare Diseases

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

IJMS, Free Full-Text

IJMS, Free Full-Text

Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study, Orphanet Journal of Rare Diseases

Subcortical Volumetric Reductions in Adult Niemann-Pick Disease Type C: A Cross-Sectional Study

Miglustat - Wikipedia

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