Parent Project Muscular Dystrophy Awards $2 Million to ACTION Network to Revolutionize Duchenne Cardiac Care

SCOTTSDALE, Ariz., June 24, 2022 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced 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.

Duchenne is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in 5,000 live male births. The absence of dystrophin in the heart contributes to a progressive deterioration of cardiac muscle, making heart failure the leading cause of death in Duchenne.

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 (DMD) 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. Ms. Furlong 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," shares Furlong. "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.

"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," says Dr. Chet Villa, MD, ACTION DMD co-chair.

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.

To learn more about PPMD's Cardiac Initiative, click here.

ABOUT PARENT PROJECT MUSCULAR DYSTROPHY

Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won five FDA approvals.

Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube. 

ABOUT ACTION

ACTION is the Advanced Cardiac Therapies Improving Outcomes Network, working together to improve critical outcomes for all children and adult congenital heart disease patients that are at risk of heart dysfunction or failure. ACTION's headquarters are located within the Heart Institute Cincinnati Children's Hospital Medical Center.  The ACTION international learning health network is led by Drs. Angela Lorts (Cincinnati Children's) and David Rosenthal (Lucile Packard Children's Hospital Stanford).

The international learning network approach allows the ability to make critical improvements faster across a collaborative system of patients, families, clinicians, researchers, and industry. As of June 2022, ACTION includes 58 pediatric hospitals in North America, as well as engagement from hospitals in 6 countries. Follow ACTION on Facebook, Twitter, Instagram, and LinkedIn.

SOURCE Parent Project Muscular Dystrophy (PPMD)