Enasidenib treatment in two individuals with D-2-hydroxyglutaric aciduria carrying a germline IDH2 mutation
Nature Medicine, Published online: 29 May 2023; doi:10.1038/s41591-023-02382-9
In a study of two children with the metabolic condition D-2-hydroxyglutaric aciduria type II, the drug enasidenib, developed as a selective mutant IDH2 inhibitor for treatment of acute myeloid leukemia, had beneficial effects on cardiac and neurodevelopmental abnormalities, indicating the potential for the repurposing of this drug for this hereditary condition.
Enasidenib treatment in two individuals with D-2-hydroxyglutaric aciduria carrying a germline IDH2 mutation
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