Recent Advances in Cardiac Therapies for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a progressive genetic disorder in which cardiomyopathy is a leading cause of mortality. In recent years, notable advances have been reported in the field of cardiac therapies for these patients. In the area of gene therapy, the MUSIC-DMD trial delivers the SERCA۲a gene via coronary infusion to evaluate its effects on myocardial performance. Regarding pharmacotherapy, phase ۲ results of Ifetroban, a thromboxane receptor antagonist, demonstrated a significant improvement in left ventricular ejection fraction. The ACTION network has also issued consensus recommendations to optimize the use of standard cardiac medications in this population. Preclinical studies of micro-dystrophin gene therapy have shown improved cardiac function in animal models, although with associated cardiac inflammation. Conversely, the cell-based therapy Deramiocel has not yet obtained FDA approval. Collectively, these findings highlight a rapidly evolving landscape of cardiac care in DMD, with the potential for combining innovative therapies with standard care to improve patient outcomes.

کلیدواژه ها:

Duchenne muscular dystrophy ، cardiomyopathy ، gene therapy ، Ifetroban ، SERCA۲a ، ACTION network