VAL-1221 for the treatment of patients with Lafora disease: study protocol for a single-arm, open-label clinical trial
Lorenzo Muccioli, Luca Vignatelli, Maria Tappatà, Serena Mazzone, Corrado Zenesini, Dustin Armstrong, DEFEAT-LD study group, Roberto Michelucci, Francesca Bisulli
BMJ Open Oct 2024, 14 (10) e085062; DOI: 10.1136/bmjopen-2024-085062