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.

Niemann-Pick disease type C

Niemann–Pick type C disease as proof‐of‐concept for intelligent biomarker panel selection in neurometabolic disorders - Papandreou - 2022 - Developmental Medicine & Child Neurology - Wiley Online Library

Treatment Options for Niemann-Pick Type C

Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression

Full article: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat

Investigation of Brain Iron in Niemann-Pick Type C: A 7T Quantitative Susceptibility Mapping Study

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

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

Treatment Options for Niemann-Pick Type C

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

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

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

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