VAL-1221 for the treatment of patients with Lafora disease: study protocol for a single-arm, open-label clinical trial

VAL-1221 for the treatment of patients with Lafora disease: study protocol for a single-arm, open-label clinical trial

Introduction Lafora disease (LD) is an ultrarare fatal progressive myoclonic epilepsy, causing drug-resistant epilepsy, myoclonus and psychomotor deterioration. LD is caused by mutations in EPM2A or NHLRC1, which lead to the accumulation of polyglucosans in the brain and neurodegeneration. There are...

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Bibliographic Details
Main Authors: Paolo Prontera, Orazio Palumbo, Massimo Carella, Corrado Zenesini, Giuseppe Damante, Laura Licchetta, Francesca Bisulli, Lorenzo Muccioli, Luca Vignatelli, Roberto Michelucci, Giuseppe D’Orsi, Elena Pasini, Maria Tappatà, Cinzia Costa, Raffaele Lodi, Serena Mazzone, Dustin Armstrong, Cosimo Altomare, Lidia Di Vito, Nicola Gambacorta, Paola Imbrici, Valentina Imperatore, Antonella Liantonio, Paola Mantuano
Format: Article
Language:English
Published: BMJ Publishing Group 2024-10-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/14/10/e085062.full

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https://bmjopen.bmj.com/content/14/10/e085062.full

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