An experimental drug may help protect heart function in people with Duchenne muscular dystrophy, a fatal genetic disorder that progressively weakens muscles. A recent study in Molecular Therapy details the findings.
Drug shows promise in preclinical models
The study tested Setanaxib, a compound designed to reduce oxidative stress, in two preclinical models of Duchenne muscular dystrophy (DMD). Treatment preserved cardiac function, limited heart enlargement, and reduced tissue scarring—key factors in preventing heart failure.
Setanaxib targets enzymes called NOX1 and NOX4, which produce reactive molecules. When these molecules accumulate, they contribute to inflammation and fibrosis. Blocking the enzymes appears to slow damage to heart tissue.
John Mably, an associate professor at the University of South Florida Health and a co-author of the study, called the results encouraging. The drug has already been tested in clinical trials for lung fibrosis and kidney and liver disease, which may speed its evaluation for DMD-related heart disease.
Heart risks in Duchenne muscular dystrophy
DMD stems from mutations that prevent the body from producing dystrophin, a protein that stabilizes muscle cells. Without dystrophin, muscles—including the heart—become vulnerable to damage from repeated contractions. Over time, healthy tissue is replaced by fat and scar tissue, weakening the heart’s ability to pump blood.
The disorder primarily affects boys because it is inherited through the X chromosome. While DMD is best known for causing mobility loss, heart complications are a leading cause of death. Treatment advances have extended lifespans, making heart protection increasingly important.
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Da-Zhi Wang, the study’s lead researcher and director of the Center for Regenerative Medicine at USF Health, stressed the need for new therapies. “Despite great progress in gene replacement therapies, Duchenne muscular dystrophy remains a devastating disease,” he said. “We must better understand the causes of DMD progression to develop treatments that ease symptoms and improve quality of life.”
The findings point to NOX4 as a potential target for future treatments. Setanaxib lowered the activity of genes linked to cardiomyopathy, a severe form of heart disease common in DMD patients.
Oxidative stress has long been linked to DMD. The disease involves complex cellular damage, and therapies addressing these mechanisms could provide broader protection than current options.
Research progress and next steps
The team, which included USF Health’s Gabriela Diniz, has studied DMD’s biological pathways for over 15 years. Their latest work offers a potential new way to preserve heart function.
Diniz noted that basic biomedical research plays a key role in understanding disease and improving healthcare. While Setanaxib’s effects in humans are still untested, its existing safety data from other trials could speed its path to DMD studies.
For now, the results support the idea that targeting oxidative stress may help manage DMD’s most dangerous complications. Future clinical trials will determine whether Setanaxib becomes a standard treatment, but the early findings suggest a new direction for managing the disease.