Treating Niemann-Pick Disease Type C

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Niemann Pick Disease - an overview

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

Niemann-Pick Type C « Niemann-Pick Children's Fund, Inc

Newborn screening test developed for rare, deadly neurological disorder – Washington University School of Medicine in St. Louis

Gene therapy shows promise for treating Niemann-Pick disease type C1

Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library

Advancements in Niemann-Pick Disease Type C Treatments

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

PDF] Niemann-Pick Type C Disease—Pathophysiology and Future Perspectives for Treatment