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Heart Transplantation and Ventricular Assist Device in Duchenne Muscular Dystrophy: A New Era.

Heart Transplantation and Ventricular Assist Device in Duchenne Muscular Dystrophy: A New Era. Pediatric transplantation Rosenthal, D. N., Amodeo, A., Butterfield, R. J., Butts, R., Chrzanowski, S., Cripe, L., Day, J., Davies, R., Duong, T., Evers, P., Gambetta, K., Harris, R., Hayes, E., Kaufman, B., Lorts, A., Mack, G., Mackenzie, S. J., Mammen, P., Markham, L., McAlister, J., McDonald, C., Nakano, S. J., Nandi, D., Navaratnam, M., Nelson, S., Parent, J. J., Piccoli, C., Richmond, M., Rocha, C., Sawnani, H., Schrader, R., Shell, R., Shih, R., Shugh, S., Smart, T., Soslow, J., Spinner, J., Taivassalo, T., Tunugtla, H., VanderPluym, C., Veerapandiyan, A., Villa, C., Wittlieb-Weber, C., Zafarullah, M., Hollander, S. 2026; 30 (1): e70253

Abstract

Duchenne muscular dystrophy (DMD) is an X-linked genetic neuromuscular disorder that is caused by a mutation in the dystrophin gene. The disease is characterized by progressive weakness of skeletal muscle, resulting in loss of ambulation and eventually respiratory insufficiency. Cardiac muscle is also involved, and cardiomyopathy is a prominent feature of DMD. The care of patients with DMD has changed in important ways over the past 15?years, with the use of chronic steroid therapy combined with noninvasive respiratory support such as continuous positive airway pressure for home use. More recently, therapies targeting specific genetic mutations in DMD have been approved, using gene-skipping oligonucleotides, and many other novel therapies are being evaluated in clinical trials, including mini-dystrophin genes delivered by viral vectors. In view of this new era in care and outcomes, it is appropriate to review the role of advanced cardiac therapies such as ventricular assist devices and heart transplantation in the care of this population. In September 2024, a group of healthcare professionals with expertise in DMD and heart failure convened to review this topic; the consensus opinion of this group (DMD Cardiac Care Consortium) is presented in this manuscript.

View details for DOI 10.1111/petr.70253

View details for PubMedID 41424087