Vitamin K Antagonist Warfarin for Palliative Treatment of Metachromatic Leukodystrophy, A Compassionate Study of Four Subjects

MLD is characterized by accumulation of sulfatides in the brain. Vitamin K regulates two enzymes in sphingolipid biosynthesis and warfarin is known to lower brain sulfatides in rats and mice. We hypothesized that warfarin may mitigate the MLD phenotype by reducing the formation of sulfatides. This c...

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Bibliographic Details
Main Authors: Mitra Assadi, Dah-Jyuu Wang, Kelly Anderson, Melissa Carran, Larissa Bilaniuk, Paola Leone
Format: Article
Language:English
Published: SAGE Publishing 2012-04-01
Series:Journal of Central Nervous System Disease
Online Access:https://doi.org/10.4137/JCNSD.S9355