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

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.

Authors

Institutions

Publication Details

Journal
Pediatric Transplantation
Published
2025-12-21
DOI
https://doi.org/10.1111/petr.70253
Primary Topic
Muscle Physiology and Disorders
Type
article
Field-Weighted Citation Impact
0.00
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

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

A. Amodeo, Carol Wittlieb‐Weber, Deipanjan Nandi, Ryan J. Butts et al.
Pediatric Transplantation
Muscle Physiology and Disorders
article

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

A. Amodeo, Carol Wittlieb‐Weber, Deipanjan Nandi, Ryan J. Butts, Katheryn Gambetta, Chet S Villa, Gordon K. Mack, Marc E. Richmond, Samuel Mackenzie, Stephen M. Chrzanowski, Stephanie J. Nakano, B. Kaufman, Christina VanderPluym, Seth A. Hollander, Manchula Navaratnam, Aravindhan Veerapandiyan, Larry W. Markham, Russell J. Butterfield, Cara Piccoli, Emily A. Hayes, Joseph A. Spinner, Craig M. McDonald, David N. Rosenthal, Ryan R. Davies, John J. Parent, Hemant Sawnani, Patrick D. Evers, Svetlana Shugh, L. Cripe, Marwa Zafarullah, Carolina Rocha, S. F. Nelson, Tina D. Duong, Renata Shih, Jennifer McAlister, Rachel Schrader, Theodore Smart, Pradeep Mammen, Angela Lorts, John Soslow, Rachel Harris, John Day, Hari Tunugtla, Richard Shell, Tanja Taivassalo
article en

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.

Pediatric TransplantationVol. 30(1)
Boston University (US), Cincinnati Children's Hospital Medical Center (US), Boston Children's Hospital (US), Arkansas Children's Hospital (US), UMass Memorial Health Care (US), Nationwide Children's Hospital (US), University of California, Los Angeles (US), Oregon Health & Science University (US), University of Utah (US), University of Florida (US), Children's Hospital Colorado (US), Monroe Carell Jr. Children's Hospital (US), Parent Project Muscular Dystrophy (US), St. Luke's Children's Hospital (US), Bambino Gesù Children's Hospital (IT), Rare Disease Therapeutics (United States) (US), Lurie Children's Hospital (US), University of Kansas Medical Center (US), Stanford Medicine (US), Texas Children's Hospital (US), University of the Sacred Heart (JP), Joe DiMaggio Children's Hospital (US), University of Rochester (US), Indiana University – Purdue University Indianapolis (US), Columbia University (US), University of Pennsylvania (US), University of California, Davis (US), The University of Texas Southwestern Medical Center (US), Vanderbilt University Medical Center (US), University of Colorado Denver (US), Stanford University (US)
Good health and well-being
Openalex Percentile: Top 37%
Muscle Physiology and Disorders
DOISourceOpenAlex
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.