PPMD is pleased to announce a $2 million grant to be awarded to the Advanced Cardiac Therapies Improving Outcomes Network (ACTION), a learning health system focused on children and adult congenital heart disease patients at risk of heart dysfunction or with heart failure.

As part of PPMD’s ongoing Cardiac Initiative, the grant will prioritize optimizing care and improving outcomes for those living with Duchenne-related cardiomyopathy and support the critical expansion of ACTION’s efforts to create the largest multicenter database on Duchenne cardiac care practices and outcomes to date.

PPMD has invested close to $7 million through its Cardiac Initiative, in the pursuit of managing and preventing cardiomyopathy (heart failure) in Duchenne. The award to ACTION comes on the heels of the organization’s first-ever Duchenne Cardiac Care Meeting in March 2022, which brought together more than 80 cardiologists and key neuromuscular providers, as well as members from the National Institutes of Health (NIH), U.S. Food and Drug Administration (FDA), and scientific and industry partners.

Led by Drs. Chet Villa (Cincinnati Children’s Hospital Medical Center), Deip Nandi (Nationwide Children’s Hospital) and Linda Cripe (Nationwide Children’s Hospital), the meeting consensus was that there is a critical need for more sufficient evidence-based data to improve the standardization of protocols and harmonization of research in real-time across networks to better understand the trajectory of Duchenne cardiomyopathy. This award to support the expansion of ACTION’s Duchenne Muscular Dystrophy project to the broader ACTION Muscular Dystrophy initiative marks a pivotal step in responding to this need

PPMD’s Founding President and CEO, Pat Furlong, announced the award during her opening address at PPMD’s 2022 Annual Conference, taking place June 23-26 in Scottsdale, Arizona:

Pat explains the critical need for this project:

“Heart issues don’t just affect some people with Duchenne; they affect ALL people with Duchenne. And while we have improved cardiac care in Duchenne, the need to find new and effective treatments that will protect and preserve heart function is urgent. PPMD’s Cardiac Initiative will be at the center of our fundraising efforts as we move forward through the second half of the year. The opportunity is here and now.

I am extremely proud to announce this $2 million award to support the expansion of ACTION’s DMD project 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.”

ACTION leverages the power of more than 50 centers throughout the United States to improve critical outcomes in pediatric heart failure, including Duchenne. In 2021 PPMD awarded Dr. Chet Villa and ACTION $150,000 for the investigation into shared decision-making and outcomes in Duchenne cardiac care as part of PPMD’s Certified Duchenne Care Center (CDCC) Inter-Institutional Grant Program.

24 centers in ACTION have been designated CDCCs, including Cincinnati Children’s and Nationwide Children’s Hospital, the member sites that house Drs. Villa, Cripe, and Nandi, the three co-chairs of the ACTION DMD subcommittee. PPMD’s CDCC program recognizes care centers that maintain the highest standards in Duchenne clinical and sub-specialty services as established by the US Centers for Disease Control and Prevention’s Duchenne Care Guidelines.

Dr. Chet Villa, MD, ACTION DMD co-chair explains: 

“Improvements in neuromuscular and respiratory care have dramatically improved outcomes in Duchenne over the last two decades. However, we have very little information about how best to care for the heart or how new medical treatments impact the heart. We can’t wait 5 or 10 years to understand this. PPMD’s support will help us get answers about how to care for the heart now. Moving forward, it will also help us understand in real-time how new therapies impact the heart and get this information to the community as soon as possible,”

PPMD’s support of ACTION’s multi-center Duchenne database also enables the collection of a real-world data set on Duchenne cardiac care practices and outcomes to date, natural history data, and expanded data collection to other dystrophinopathies. The use of an electronic platform reduces barriers to entry. It allows providers to engage as many families as possible independent of geography, to better understand the impact of therapies on quality of life. The database will facilitate a data-driven approach to consistent patient/provider education, best practices, and future clinical trial design needed to move the field forward and change lives.

PPMD’s Research Strategy & Investments

Ending Duchenne means attacking the disease with multiple approaches from multiple angles and with your support we will continue to not only explore the most promising treatments, we will do it while protecting the hearts and muscles of our loved ones. To learn more about our robust Research Strategy, funding initiatives and strategies for accelerating drug development, click here.