What Is Niemann-Pick Disease?

Niemann-Pick disease symptoms vary. The disease includes a group of conditions characterized by accumulation of sphingomyelin in cells of the body.

Immune dysfunction in Niemann‐Pick disease type C - Platt - 2016 - Journal of Neurochemistry - Wiley Online Library

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

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

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

White and gray matter alterations in adults with Niemann-Pick disease type C

Niemann-Pick disease type C

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

Niemann–Pick disease, type C - Wikipedia

Niemann-Pick disease