Empagliflozin in Lafora disease: A first-in-human 6-month pilot study with electroclinical and FDG-PET findings.
OBJECTIVE Lafora disease (LD) is a rare, fatal neurodegenerative epilepsy with no approved treatments. Empagliflozin, a sodium-glucose cotransporter-2 (SGLT2) inhibitor, has shown neuroprotective actions in preclinical models through modulation of glycogen metabolism, induction of a pseudo-ketogenic state, improved mit...