Cara A Timpani, Stephanie Kourakis, Danielle A Debruin, Dean G Campelj, Nancy Pompeani, Narges Dargahi, Angelo Patrick R Bautista, Ryan M Bagaric, Elya J Ritenis, Lauren Sahakian, Didier Debrincat, Nicole Stupka, Patricia Hafner, Peter G Arthur, Jessica R Terrill, Vasso Apostolopoulos, Judy B De Haan, Nuri Gueven, Dirk Fischer, Emma Rybalka
New medicines are urgently required to treat the fatal neuromuscular disease, Duchenne muscular dystrophy (DMD). Dimethyl fumarate (DMF) is a potent immunomodulatory small molecule nuclear erythroid 2-related factor 2 (Nrf2) activator with current clinical utility in the treatment of multiple sclerosis and psoriasis that could be effective for DMD and rapidly translatable. Here, we tested two weeks of daily 100mg/kg DMF versus 5mg/kg standard care prednisone (PRED) treatment in juvenile mdx mice with early symptomatic DMD...
September 26, 2023: JCI Insight