Parent Project Muscular Dystrophy (PPMD) funds ACTION to expand Muscular Dystrophy collaboration efforts

ACTION receives $2 million grant from Parent Project Muscular Dystrophy (PPMD) to expand muscular dystrophy focused research and quality improvement initiatives

CINCINNATI, Ohio — Parent Project Muscular Dystrophy (PPMD) has announced that they will be awarding a $2 million grant to ACTION’s muscular dystrophy collaborative.

“The heart is a muscle too, and finding new and effective treatments to preserve the heart muscle has been a constant theme for PPMD. This award to support the continued growth and expansion of ACTION’s Duchenne efforts is critical to driving care forward through clinical trials, continued cardiac research, and more.”

Pat Furlong, PPMD Founding President & CEO

Advanced Cardiac Therapies Improving Outcomes Network (ACTION) is a learning health system focused on 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 at 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).

This ACTION Duchenne Muscular Dystrophy (DMD) collaborative is led by Drs. Chet Villa (Cincinnati Children’s), Deip Nandi (Nationwide Children’s Hospital), and Linda Cripe (Nationwide Children’s Hospital).

DMD is caused by the lack of a functional dystrophin protein and leads to progressive skeletal, lung, and cardiac muscle weakness. The long-term survival of those living with DMD is increasing, leading to a growing number of people living with heart dysfunction. As a result, clinicians and families are having to make significant decisions regarding cardiac care. This includes when to start cardiac medications and when to consider advanced therapies such as cardiac devices or transplantation.

“These practices are rapidly evolving and there is an urgent need to understand how best to treat cardiac disease in order to improve the outcomes for these patients.”

Chet Villa, MD, ACTION, Cincinnati Children’s

PPMD’s initial investment in the ACTION DMD committee in 2021 is improving the care of DMD cardiomyopathy patients and helped us to identify the need for expansion to other diseases caused by low or non-functional dystrophin. These include Becker muscular dystrophy (BMD) and females with dystrophinopathy. The increased PPMD funding will provide ACTION’s clinicians, researchers, parents, and patients the resources to study and understand cardiac disease and outcomes in all dystrophinopathies. This information will be used to guide and standardize cardiac care and improve outcomes for patients at more than 50 ACTION sites worldwide.

PPMD’s gift will allow the expansion of ACTION’s DMD committee’s initiatives including: 

  • DMD Database Expansion
  • Increase institution participation and ensure diverse representation
  • Establish use of real-world data and prospective clinical trials for treatment of cardiac disease in muscular dystrophy
  • Develop ACTION Patient Reported Outcomes (PRO)
  • Develop educational modules for patients, caregivers, and providers

In addition to the data collection and quality improvement initiatives, ACTION’s education site, My ACTION Education, will be expanded to include muscular dystrophy related materials for patients, caregivers, and providers. The site features engaging animations, infographics and articles that help children, young adults, parents, and caregivers better understand heart failure and explore their treatment options.

Together, we are moving the field forward and bringing hope to thousands of patients living with muscular dystrophy.

Media contact: Rosie Ecker
For more information, contact: Nikita D’Souza

See the Parent Project Muscular Dystrophy full press release here.