Searching for a cure for Niemann-Pick Type C - NemaMetrix

Niemann-Pick Type C is a rare and fatal disorder affecting children. We explore the efficacy of new potential therapeutics with the ScreenChip System.

Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C

PDF) Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-Β-Cyclodextrin

Niemann-Pick disease type C

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 type C Mnemonic

Searching for a cure for Niemann-Pick Type C - NemaMetrix

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

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

Niemann-Pick Type C Disease

Psychiatric symptoms as a harbinger of adult-onset Niemann-Pick Type C - ScienceDirect