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Targeting layered regenerative inflammation tissue zones in dystrophic muscle

Duchenne Muscular Dystrophy (DMD) is a serious and deadly disease that primarily affects children. It is a degenerative disease that leads to the loss of muscle tissue, which is replaced by fibrosis and fat, resulting in significant mobility impairments. There is currently no cure for DMD, but there are drug and gene therapy efforts to convert the disease to a milder form. One major challenge is finding effective treatments that can reduce inflammation without suppressing the immune system and prevent or reverse muscle loss. One novel approach we have been studying is to modify immune cells, particularly macrophages, to promote regeneration and the formation of new tissue. This project aims to evaluate the impact of various anti-inflammatory treatments on the formation of regenerative tissue zones that we found to be sensitive to glucocorticoid treatment. The study will also focus on the therapeutic efficacy of specific treatments that promote a pro-repair macrophage phenotype and restore the regenerative capacity of dystrophic muscle in aging DMD patients. Overall, in light of our recent compelling findings, the proposed work will re-evaluate the impact of current DMD standard-of-care drugs on muscle regeneration using state-of-art aproaches and has the potential to introduce new combination therapies for muscular diseases to support the growth of muscle tissue.
Grantee: Andreas Patsalos, Ph.D.
Grant type: Development Grant
Award total: $210,000.00
Institution: Johns Hopkins University School of Medicine
Country: United States