by: Parent Project Muscular Dystrophy
Someone says, “wait and see,” while someone else says, “start medication early.” Someone recommends cardiac MRI, while someone else suggests, “echo is probably fine.”
All the while, new technology abounds, new drugs come to market, and it leaves us feeling desperate – for an answer to our questions, for understanding, for a plan for today and the future. We wonder, we ruminate: Is the heart built to last?
The heart is a muscle TOO, and finding new and effective treatments to preserve the heart muscle has been a constant theme for PPMD.
PPMD recently held a meeting to focus exactly on these issues – what we know, what we need to learn, how we will know what works, and what are the best ways to preserve heart function. It is clear, we need to develop a plan so that we can move forward as fast as possible.
Convening 80+ Expert Cardiologists & Professionals
PPMD’s Duchenne Cardiac Care Meeting was led by Drs. Chet Villa (Cincinnati Children’s Hospital), Deip Nandi (Nationwide Children’s Hospital) and Linda Cripe (Nationwide Children’s Hospital). Nationwide and Cincinnati are both member sites of the Advanced Cardiac Therapies Improving Outcomes Network (ACTION), a collaborative that includes cardiologists from around the world focused on heart failure in kids. Drs. Villa, Nandi, and Cripe have been focused on Duchenne for many years and believe the heart failure seen in Duchenne is far different than other types of heart failures in children.
Drs. Villa, Nandi, and Cripe have been working with ACTION to set up a dedicated arm within that network to focus efforts entirely on Duchenne and ensure it becomes a critical area within the network. Their goal is to dive deep to fully understand heart disease in Duchenne, how the heart cells are replaced by scar tissue and fat, and how that relates to changes in the pumping action of hearts.
With this arm already in place, it was clear we needed to support and expand upon that pre-existing arm to evolve to serve as a network specific for Duchenne and bring people together to drive care forward through clinical trials, continued cardiac research, and more. As a result, PPMD’s Duchenne Cardiac Care Meeting was developed to bring together more than 80 cardiologists from institutions around the country, people from the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA), and scientific and industry partners to discuss:
- What do we know about the heart?
- Do people with Duchenne have abnormal heart rhythms?
- What testing is being used by each center?
- What are centers doing that are the same, or different?
- Which testing gives the most detailed information?
- What medicines are being used?
- What other heart failure drugs might be effective in the Duchenne population?
- What about new drugs on the horizon and their ability to support the Duchenne heart?
Taking Action on What is Next
This meeting was incredibly valuable to convene specialists and share learnings, and most critically, we now have our marching orders for the work that needs to be done next. The need is urgent. The message is clear. We need to support the continued growth and expansion of ACTION’s Duchenne efforts to develop a standard of care for testing and care that is consistent across the United States – and the world – to allow us to learn together how best to protect every single beat, evaluate potential new therapies, and do our very best to keep the heart stronger, longer.
Your Voice is Critical
PPMD’s Annual Conference is coming in June, and we hope to see you there! As part of this community gathering, we are planning the first ever Duchenne Cardiac Care Town Hall, part of an extended 3-hour session dedicated specifically to Duchenne hearts.
We want to share with you what the experts are learning, but more importantly, we want to LISTEN and HEAR from you about your worries, questions, hopes, dreams for Healthy Hearts so that together, we can forge a path forward to create meaningful treatments and change lives.
Register Now >
