Announced in March 2024, Xuhui Liu, MD (co-director of the Feeley-Liu Lab) was awarded a Translational Research Award for Duchenne Muscular Dystrophy Research Program from Congressionally Directed Medical Research Programs (CDMRP) of the Department of Defense. The grant will be used to investigate the role of a beta-3 adrenergic receptor agonist in improving the cardiac and respiratory function of  Duchenne muscular dystrophy (DMD) in a mouse model.

Duchenne muscular dystrophy (DMD) is a severe, progressive, muscle-wasting disease that affects around 1 in 3500 to 1 in 5000 live male births. The most impaired muscle in DMD patients is the diaphragm, and its wasting is responsible for respiratory failure. More than 90% of DMD patients are affected by some degree of cardiomyopathy. With optimal care, most patients with DMD die between 20 and 40 years of age from either cardiac or respiratory failure. Thus, improving cardiac/respiratory function is critical to improving DMD patient survival and quality of life.

In this awarded study, Dr. Liu will test the role of Mirabegron, an FDA-approved beta3 adrenergic receptor agonist in treating cardiac/respiratory dysfunction in a preclinical DMD mouse model with severe heart and diaphragm fibrosis and dysfunction. Most patients diagnosed with DMD are on glucocorticoid as a first-line treatment. Thus Dr. Liu will also test the effect of combined treatment of Mirabegron and glucocorticoid in this study. Because Mirabegron is an FDA-approved drug with an excellent safety profile, essential preclinical data gained from this study may lead to an expedited transition of clinical trials and benefit DMD patients in the near future.

The Feeley-Liu lab is looking for enthusiastic and motivated scientists/research associates to join our research group. Interested candidates can send their CVs to [email protected]