Treatment Options for Niemann-Pick Type C

Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular Diagnostics Section of the Genetic Laboratory at Rush Medical College in Chicago, describes dif

IJMS, Free Full-Text

FDA says Hold Off on Zavesca (Miglustat) approval. « Niemann-Pick Children's Fund, Inc

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

Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016

Niemann-Pick Type C (NPC) Market Outlook and Forecast - Thelansis

Imaging of neuroinflammation in adult Niemann-Pick type C disease

Niemann Pick Disease - Rivin

Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression

Adult Onset Niemann-Pick type C (NPC) – NNPDF

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