Empagliflozin Repurposing for Lafora Disease: A Pilot Clinical Trial and Preclinical Investigation of Novel Therapeutic Targets

Background: Lafora disease (LD) is an ultra-rare and fatal neurodegenerative disorder with limited therapeutic options. Current treatments primarily address symptoms, with modest efficacy in halting disease progression, thus highlighting the urgent need for novel therapeutic approaches. Gene therapy...

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Bibliographic Details
Main Authors: Giuseppe d’Orsi, Antonella Liantonio, Paola Imbrici, Nicola Gambacorta, Giorgia Dinoi, Cosimo Damiano Altomare, DEFEAT-LD Study Group, Massimo Carella
Format: Article
Language:English
Published: MDPI AG 2025-05-01
Series:Methods and Protocols
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Online Access:https://www.mdpi.com/2409-9279/8/3/48
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