Expanding Access to Advanced Neuromuscular Therapies: Q&A With Chet Villa, MD
The Neuromuscular Cardiology Program, part of the Heart Institute at Cincinnati Children’s, treats children with complex medical needs, including Duchenne muscular dystrophy and heart disease.
As one of the largest and most distinguished international programs, experts from nearly 10 subspecialties—including cardiology, neuromuscular, pulmonology and orthopaedics—partner to provide top-level multidisciplinary care.
In the past five years, Neuromuscular Cardiology has seen significant growth and increased patient volume. Chet Villa, MD, pediatric cardiologist and a lead physician in the Neuromuscular Cardiology Program, shares insights about recent referrals and research initiatives.
How Has the Program Grown in the Past Few Years?
Since 2021, we’ve seen significant program growth across this multispecialty clinic, as well as cardiology.
Why are People Seeking Out this Program at Cincinnati Children’s?
People come here for clinical care so that they can access the most advanced clinical trials. We also have strong subspecialties. When families meet with us, especially when their child has a rare disease, they don’t have to see two people here, three people at home and one person in Georgia. At Cincinnati Children’s, they see all subspecialties at once. It allows them to condense their visits because of the strength of the overall program.
We also perform rare procedures, including ventricular assistive device (VAD) surgery in children with neuromuscular disease. I work in both the VAD Program and the Neuromuscular Cardiology Program, which is uncommon because it’s really two subspecialties.
What are Current Neuromuscular Cardiology Research Initiatives?
We’re leading a few cardiac-focused, multi-center gene therapy trials—one for Friedreich’s ataxia and one for Duchenne muscular dystrophy. We’re one of only three centers around the country offering novel therapies like gene therapy for Duchenne. Similarly, we’re one of the only centers looking at gene therapies for Friedreich’s ataxia from a pediatric perspective.
Our ACTION muscular dystrophy trial is the largest prospective study of cardiac disease in people with Duchenne in the world. We’ve enrolled over 1,200 patients so far, which is a gigantic number, and have had multiple related publications, including:
- Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy
- Initial Multicenter Experience with Ventricular Assist Devices in Children and Young Adults with Muscular Dystrophy: An ACTION Registry Analysis
How Does Research Intersect with Clinical Care?
We aim to integrate our research fully with the clinical care patients receive. Our program rolls out clinically what we learn in our research programs quicker, faster and more effectively than most pediatric institutions.
For example, our research into what happens at the tissue level is changing how we care for patients with Duchenne muscular dystrophy. This work showed that if we wait to start heart medications, we may miss a window to prevent scar tissue forming in the heart. We’re now starting to use medications earlier to try to prevent scar tissue from forming.
We’ve also pioneered ways for nurses to coordinate care with our physicians to help patients and families get both new and existing cardiac medications to the right dose safely and quickly.
Dr. Villa is a pediatric cardiologist who specializes in heart failure and transplantation. He and his team care for children with complex medical needs and heart disease. To contact them, call 513-803-3000.
(Published November 2025)
