Searching for a cure for Niemann-Pick Type C - NemaMetrix
Por um escritor misterioso
Descrição
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 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
Why is Niemann-Pick Type C Difficult to Treat?
Investigating the Mechanism of Cyclodextrins in the Treatment of Niemann‐Pick Disease Type C Using Crosslinked 2‐Hydroxypropyl‐β‐cyclodextrin,Small - X-MOL
PDF) Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-Β-Cyclodextrin
Niemann-Pick Type C Disease
Treatment Options for Niemann-Pick Type C
Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology
Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression
Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression
New murine Niemann-Pick type C models bearing a pseudoexon-generating mutation recapitulate the main neurobehavioural and molecular features of the disease
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