N-Octyl-α-4-epivalienamine is an orally active and CNS-penetrant molecular chaperone that induces high expression of the deficient β-galactosidase activity. N-Octyl-α-4-epivalienamine ameliorates symptoms and increase survival rate in a mouse model of G M1 -gangliosidosis. N-Octyl-α-4-epivalienamine can be used for neurogenetic disease research .
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