TAMPA, Fla. (July 17, 2026) – Researchers at the University of South Florida USF Health Morsani College of Medicine have identified a potential pathway that could protect cardiac function in people with Duchenne muscular dystrophy, a progressive and fatal genetic disease that weakens the body’s muscles.
Published in Molecular Therapy , the new study examined the effects of the experimental drug Setanaxib in two preclinical models of DMD. Researchers found that the treatment preserved cardiac function, limited heart enlargement and reduced tissue scarring.
The findings also suggest that an enzyme known as NOX4 could serve as a potential target for future treatments for cardiomyopathy, a serious form of heart disease associated with DMD.
The study was led by Da-Zhi Wang, professor in the USF Health Morsani College of Medicine and director of the Center for Regenerative Medicine at the USF Health Heart Institute . The research team also included USF Health’s John Mably , associate professor, and Gabriela Diniz , assistant professor, as well as outside collaborators.
“Despite great progress in the development of gene replacement therapies, Duchenne muscular dystrophy remains a devastating disease,’’ Wang said. “Understanding the causes of DMD progression is urgently needed to advance therapies that alleviate symptoms and improving quality of life.”
DMD primarily affects boys because it is inherited through the X chromosome. The disease is best known for causing progressive weakness in the muscles used to walk and move. But one of the disease’s most serious effects occurs in the heart.
The condition is caused by mutations that prevent the body from producing functional dystrophin, a protein that helps stabilize and protect muscle cells.
Without dystrophin, muscle cells become more vulnerable to damage from repeated contractions. Over time, healthy muscle can be replaced by fat and stiff scar tissue.
The heart is especially vulnerable because it must contract continuously to circulate blood throughout the body. As cardiac damage accumulates, the heart can enlarge and lose its ability to pump effectively, eventually leading to heart failure.
Treatments have helped people with DMD live longer, making the protection of the heart an increasingly important part of managing the disease.
In the new study, researchers evaluated Setanaxib, an experimental drug designed to limit a damaging process known as oxidative stress.
The drug targets NOX1 and NOX4, enzymes that produce highly reactive molecules within cells. At normal levels, these molecules play useful roles in the body; however, when too many are produced, they can contribute to inflammation and fibrosis.
Researchers found that Setanaxib helped preserve the heart’s ability to pump blood while reducing inflammation and fibrosis. It also lowered the activity of genes associated with cardiomyopathy.
The results suggest that blocking this pathway may help slow the progression of DMD-related heart disease.
“The results are very promising,” Mably said. “The Nox4 inhibitor has already been tested in clinical trials for the treatment of lung fibrosis and kidney and liver disease. We hope it will also be tested soon in clinical trials in DMD patients to slow the progression of heart disease.’’
The findings build on more than 15 years of DMD research in Wang’s laboratory. During that time, the team has sought to better understand how the disease progresses and identify biological mechanisms contributing to the development of new therapies.
“Our new study represents an important step forward for the field and for our cardiac and regenerative medicine program,” Diniz said. “It shows how efforts in basic biomedical sciences are crucial for understanding human disease and health care advancements.”
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About the University of South Florida
The University of South Florida is a top-ranked research university serving approximately 50,000 students from across the globe at campuses in Tampa, St. Petersburg and USF Health. In 2025, U.S. News & World Report recognized USF with its highest overall ranking in university history, as a top 50 public university for the seventh consecutive year and as one of the top 15 best values among all public universities in the nation. U.S. News also ranks the USF Health Morsani College of Medicine in the highest tier, placing it as one of the top 16 medical schools in the nation and inside the top 10 among public universities. USF is a member of the Association of American Universities (AAU), a group that includes only the top 3% of universities in the U.S. With an all-time high of $750 million in research funding in 2025 and as a top 20 public university for producing U.S. patents, USF uses innovation to transform lives and shape a better future. The university generates an annual economic impact of nearly $10 billion for the state of Florida. USF’s Division I athletics teams compete in the American Conference. Learn more at www.usf.edu .
Molecular Therapy
Experimental study
People
Treatment with the Nox1/4 inhibitor Setanaxib ameliorates cardiac function in mouse models of Duchenne muscular dystrophy
26-Jun-2026