Cardioprotection and Heart Failure Management in Duchenne and Becker Muscular Dystrophies: A State-of-the-Art Review Across Pediatric and Transitional Age

Advances in respiratory care have significantly extended the life expectancy of patients with Duchenne and Becker muscular dystrophies (DMD/BMD), making progressive dilated cardiomyopathy the leading cause of mortality in this population. Historically, a conservative “watch and wait” approach was often maintained until overt systolic dysfunction became evident. However, the advent of advanced echocardiographic technologies, particularly Global Longitudinal Strain (GLS) imaging, has demonstrated that myocardial impairment begins in early childhood, long before a decline in ejection fraction occurs. This critical evidence strongly advocates for a paradigm shift toward early, proactive cardioprotective strategies. As patients progress into the transitional age and adulthood, the pediatric prophylactic approach (traditionally based on ACE inhibitors and beta-blockers) must integrate with evolving adult heart failure guidelines. This comprehensive review critically evaluates the pathophysiological continuum of dystrophic cardiomyopathy. We highlight the prognostic value of early subclinical detection (GLS) and specifically discuss the transition to modern guideline-directed medical therapies (GDMTs), exploring the emerging role of Angiotensin Receptor-Neprilysin Inhibitors (ARNI, e.g., sacubitril/valsartan) and SGLT2 inhibitors (e.g., dapagliflozin) in this specific cohort. Finally, we address the role of advanced device therapies (ICD, CRT, and left ventricular assist devices) in end-stage disease. By bridging pediatric neurology and adult cardiology, this review aims to equip clinicians with an updated, multidisciplinary framework to optimize cardiovascular outcomes in the growing population of transitional-age DMD/BMD patients.