Due to their previous work regarding the relationship between FAPs and muscle dystrophy, the Feeley-Liu Lab was awarded a $300,000 research grant by the Muscle Dystrophy Association (MDA) to pilot a study that examines mirabegron as a possible therapy for those affected with muscle dystrophy.

Duchenne muscular dystrophy (DMD) is a fatal disease affecting around 1 in 3500 to 1 in 5000 live male births. Boys with DMD usually lose the ability to walk and feed in their teens and die from respiratory insufficiency or cardiomyopathy in early adulthood. There is no cure for this disease at this time. In our previous studies, we found that a group of muscle stem cells, named Fibro/adipogenic progenitors (FAPs) can differentiate into brown fat-like cells to prevent muscle degeneration and promote muscle regeneration, under the stimulation of a group of drugs, called beta3 adrenergic receptor (B3AR) agonists. Mirabegron is an FDA-approved B3AR agonist currently used for treating patients with hyperactive bladders, including kids. In our pilot study, we have shown that Mirabegron significantly improved the health and extended the life span of mice with DMD. In this study, we will determine the optimal dose of Mirabegron in treating DMD in a preclinical DMD mouse model. We will also define the role of Mirabegron in reducing heart and skeletal muscle fibrosis, a critical pathological change that leads to heart failure and muscle weakness. Results from this study will provide essential preclinical data that allows the filing of clinical trials application of Mirabegron for treating DMD. Considering the fact that Mirabegron is an FDA-approved drug, the successful accomplishment of this study may lead to an expedited transition of clinical use of Mirabegron on DMD patients.