| First Author | Timpani CA | Year | 2023 |
| Journal | JCI Insight | Volume | 8 |
| Issue | 21 | PubMed ID | 37751291 |
| Mgi Jnum | J:343612 | Mgi Id | MGI:7566685 |
| Doi | 10.1172/jci.insight.165974 | Citation | Timpani CA, et al. (2023) Dimethyl fumarate modulates the dystrophic disease program following short-term treatment. JCI Insight 8(21) |
| abstractText | 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 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 2 weeks of daily 100 mg/kg DMF versus 5 mg/kg standard-care prednisone (PRED) treatment in juvenile mdx mice with early symptomatic DMD. Both drugs modulated seed genes driving the DMD disease program and improved force production in fast-twitch muscle. However, only DMF showed pro-mitochondrial effects, protected contracting muscles from fatigue, improved histopathology, and augmented clinically compatible muscle function tests. DMF may be a more selective modulator of the DMD disease program than PRED, warranting follow-up longitudinal studies to evaluate disease-modifying impact. |
