New treatment for pyridoxine-dependent epilepsy due to ALDH7A1 deficiency: first proof-of-principle of upstream enzyme inhibition in the mouse

Authors van Karnebeek Clara D M, Gailus-Durner Valérie, Engelke Udo F, Seisenberger Claudia, Marschall Susan, Dragano Nathalia R V, da Silva-Buttkus Patricia, Leuchtenberger Stefanie, Fuchs Helmut, Hrabě de Angelis Martin, Wevers Ron A, Coughlin Curtis R, Lefeber Dirk J
Journal Brain Communications
Publication Date 2025-10-30
DOI 10.1093/braincomms/fcaf397

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