Meeting report: Expanding access to advanced cardiac therapies, including ventricular assist devices (VADs) and heart transplantation in muscular dystrophy.

Cardiomyopathy remains a leading cause of mortality in Duchenne muscular dystrophy (DMD), yet advanced therapies such as ventricular assist devices (VAD) and heart transplantation are rarely considered. In September 2024, a multidisciplinary group of neuromuscular specialists, cardiologists, intensivists, anesthesiologists, surgeons, respiratory therapists, rehabilitation specialists, and patient advocates convened to challenge the clinical challenges and institutional barriers that have historically excluded individuals with DMD from these life-prolonging interventions. The charge of the meeting was to develop a practical roadmap to guide the timely referral, evaluation, and management of DMD patients with advanced heart failure. Presentations focused on the evolving treatment landscape and reviewed limitations of standard heart failure assessments-including ejection fraction and symptomatology-while exploring alternative evaluation tools such as cardiac MRI, biomarkers, adapted exercise testing, and patient-reported outcomes. Discussions addressed candidacy, surgical considerations, respiratory and nutritional optimization, and psychosocial supports. Participants determined that VAD and transplant should be offered to appropriate candidates and began collaborative development of clinical protocols, which were then completed and published online approximately one year after the meeting concluded.