November 14, 2023 / Care,Research

Advancing Duchenne Cardiac Care: A Year of Impact and Gratitude

Cardiac care is at the heart of Duchenne, which is why it remains a critical element of PPMD’s efforts to optimize care and research through our ongoing Cardiac Initiative. As we look back on the achievements of 2023 and ahead to 2024, we extend our heartfelt gratitude to our donors. The impact of your support is felt in each milestone, a testament to our collective fight for every heartbeat. Together, we are making a difference, convening experts and driving the innovation needed to ensure we protect the heart.


Cardiac Workshops:

Building on the momentum from our 2022 Cardiac Workshop in Charleston, South Carolina, PPMD kicked off 2023 by sharing insights and findings with the community through a comprehensive meeting report. A vital precursor to our 2023 Cardiac Workshop, this report set the stage for the collaborative efforts that followed.

In March 2023, PPMD’s care team brought together more than 80 cardiologists and cardiac researchers in New Orleans, LA for the second workshop. Delving deeper into cardiac care, these experts explored critical topics including gene therapy’s impact on the heart, carrier care, use of newly approved medications, how data answer research questions, and identifying goals for the field to move the needle in cardiac care. A detailed meeting report is currently in preparation, ensuring the broader community benefits from the shared learnings. 

Recognizing the ongoing need for dialogue, plans are already underway for the Cardiac Workshop III in May 2024 in Baltimore, Maryland. This workshop will focus on the development of adult cardiac care considerations, Becker muscular dystrophy, and post-gene therapy cardiopulmonary monitoring. 

Investments in Research:

Furthering our investment in cardiac care and supporting the development and understanding of critical cardiac biomarkers, PPMD announced a $65,000 investment to support the ongoing work of Dr. Jonathan Soslow of Vanderbilt University, along with his collaborators Dr. Larry Markham (Riley Children’s Hospital) and Dr. Chris Spurney (Children’s National Medical Center). Dr. Soslow’s work will aim to deepen our understanding of cardiomyopathy and develop predictive biomarkers.

ACTION Learning Network:

Thanks to the support of our donors at the end of 2022, PPMD committed $2 million over three years to support PPMD’s Cardiac Initiative and the ACTION Learning Network, a collaborative network of physicians from across the US and Canada. We are pleased to see continued progress stemming from our funding commitment including a newly released toolkit of resources, designed to empower and inform families navigating Duchenne. The consortium has also developed a post-gene therapy harmonization document, which aims to offer guidance and consistency in the monitoring and treatment of individuals with Duchenne post-gene therapy administration. 

A Heartfelt Thank You

Thank you again for your continued support of PPMD and our ongoing Cardiac Initiative. Your generosity not only fuels our progress in the fight to end Duchenne but also plays a crucial role in ensuring hearts grow stronger with every beat.

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