lithium gluconate + glyceryl triacetate

Lithium gluconate + glyceryl triacetate is an investigational oral combination therapy studied primarily for Canavan disease, a rare autosomal recessive neurodegenerative disorder caused by mutations in the ASPA gene. Lithium gluconate is a salt form of lithium, which acts as an inhibitor of glycogen synthase kinase-3 (GSK-3), influencing neurodevelopmental and metabolic pathways. Glyceryl triacetate (also known as triacetin or GTA) is a short-chain triglyceride that serves as an acetate precursor; it increases brain acetate levels to support myelin lipid synthesis, addressing the deficiency seen in Canavan disease. The combination aims to provide both neuroprotective effects via GSK-3 inhibition and metabolic support through enhanced acetate availability for myelination. Clinical studies have evaluated the safety and tolerability of this combination in patients with Canavan disease[1][2][6].

Other names

lithium gluconate and glyceryl triacetatelithium gluconate + GTAlithium gluconate (drug) glyceryl triacetate GTA (drug)