Abstract:Background
Vanishing white matter disease (VWMD) is a rare leukodystrophy caused by mutations of the guanine exchange factor eIF2B that typically presents with juvenile onset. There are few treatments and no cures for the disease. Recent progress in the field has established mitochondrial dysfunction and endoplasmic reticulum (ER) stress to be strongly implicated in observed glial cell pathology. Drug repurposing offers a rapid approach toward translation of therapeutics with already-licensed drugs.
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