Last week, PPMD held the 2024 Cardiac Care Workshop in Baltimore, Maryland, which included 40 academics and professionals and representatives from 11 industry partners to discuss how to move beyond the current state of cardiac care to accelerate research, advance standards of care, and better understand Duchenne and Becker in a rapidly evolving and complicated landscape. 

As part of PPMD’s Care Consensus Meeting Series, PPMD convenes leading experts to address critical components in the management of Duchenne and Becker. Hosted by PPMD and expert committees, these meetings identify and assess key gaps in care, education, and understanding the complexities of the condition. 

This third meeting in PPMD’s three-part Cardiac series, attendees took a deeper dive into the rapidly evolving landscape of approved therapy and post-gene therapy cardiac monitoring and lifespan cardiac care. Topics this year included pathology and imaging, cardiac monitoring in clinical trials, gene therapy, transplantation, and more. 

Setting the Stage

Pat Furlong, PPMD’s Founding President and CEO, and James Wilson, MD, PhD, Director of the gene therapy program and the Rose H. Weiss Professor and Director of the Orphan Disease Center at the University of Pennsylvania, set the tone of the meeting presenting critical questions and needs within the community, highlighting  areas of need around expedited models by which to accelerate data collection and drug development, and how to better understand changing trends in care for individuals treated with gene therapy. 

New Data, New Treatment Paradigm

In the workshop’s first session, emerging data from ACTION-DMD was presented, including data around pathology, imaging, and medication use. This led to an engaging discussion around when to start cardiac medications, which medications to use, and acknowledgement that it is  time to move beyond currently published care standards and consider initiating cardiac medicines before the age of 10.

Engaging Industry

Linda Cripe, MD led a panel discussion with industry partners from across the Duchenne space, including gene therapy, next-general exon skipping drugs, and small molecule drugs to better understand limitations and challenges to including cardiac outcomes in clinical trials. This allowed for mutual learnings for both industry and clinicians on how they can partner together to better incorporate consistent cardiac outcomes into clinical trial development in an effort to understand the cardiac implications of these therapies. . 

Gene Therapy

Workshop attendees then discussed  gene therapy and cardiac implications post-treatment, exploring how to leverage existing networks to both standardize and better understand how gene therapy impacts cardiac function. 

Transplantation in Dystrophinopathy

Shifting away from medical therapies and gene therapy, the group then engaged in a timely discussion around transplantation in dystrophinopathy, including ethical implications and a charge to reconsider how we think about heart transplantation and develop guidelines to encourage clinicians to think proactively about VAD and transplantation for their patients. .

No Data, No Paradigm

Data from ACTION enabled a discussion focused on gaps in cardiac data as well as models by which we can better understand new and emerging research and leverage networks like ACTION to standardize how we collect the data, allowing us to do better, learn more, and optimize cardiac care. 

Moving Forward

To close the workshop, Chet Villa, MD, Jonathan Soslow, MD, Deipanjan Nandi, MD, Linda Cripe, MD and Larry Markham, MD led meeting participants into a  discussion around how we can take learnings from the Cardiac Workshop Series into a  new world of cardiac care, post-gene therapy care, and leverage networks like ACTION and the DMDCCC (DMD Cardiovascular Care Consortium) to create a clinical trials collaborative to expedite the establishment of updated care guidelines, development of new therapies, and improve monitoring and care of individuals post-gene therapy. 

Next Steps

We are eager to see the impact of PPMD’s Cardiac Care Workshop series over the last three years. With a clear vision moving forward we hope to create a path ahead for aggressive, proactive cardiac care for all individuals living with dystrophinopathy. 

To hear more about the Cardiac Care Workshop, join us at PPMD’s 30th Annual Conference, taking place June 27-29, 2024 in Orlando, Florida. We hope to see you there – register today to join us in person or virtually!! We look forward to sharing more detail about this meeting with the community, including an opportunity to engage and ask questions.