Searching for a cure for Niemann-Pick Type C - NemaMetrix
Por um escritor misterioso
Last updated 10 abril 2025

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

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

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

Niemann-Pick disease type C: Video & Anatomy

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

IJMS, Free Full-Text

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

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

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

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

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

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