Early data from 500 males in the ACTION dystrophinopathy registry show substantial cardiac involvement and broad use of cardioprotective therapies. Ongoing follow-up will define the modern natural history of cardiomyopathy in dystrophinopathy.
We hope you are enjoying your summer vacations and finding time to rest and rejuvenate with your families! ACTION has been busy with new publications, tools, resources, and projects (see links and features below). We are thrilled to announce the launch of our new VAD Adverse Event Definitions! These new and updated definitions will help us capture events more accurately and efficiently. The DCC also streamlined fields, improved forms, and added instructional text throughout to make data entry easier and faster. Thanks to all those who worked on this important project! Lastly, we are now taking proposals for ISHLT 2026, see more details below on the submission process.
The Pulse — Aug 2025 Read More »
In a prospective study of 265 males with Duchenne Muscular Dystrophy, nearly 30% of those with moderate or severe heart dysfunction were not receiving full consensus-directed cardiac therapy, and most did not reach target medication doses—highlighting the need to optimize treatment strategies as cardiac disease becomes a leading cause of death in DMD.
Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy Read More »
An expert opinion statement, addressing a critical gap in cardiac care for Duchenne muscular dystrophy – providing thorough recommendations for the initiation and titration of cardiac medications based on disease progression and patient response.
This multi-center retrospective pilot study evaluated the current state of HF medication therapy in children and found that, despite a robust evidence base in adults and recommendations for children, use of ACEi/ARB/ARNi, BB, and MRA is not universal in children with EF <40%; indeed, less than half of children received medications from all three classes by 6 months of follow-up...
Check out our educational handout on An Introduction to Myocarditis. Included is information about: What is Myocarditis? How is it diagnosed? What are the causes and the symptoms? Treatment options
An Introduction to Myocarditis Read More »
Check out ACTION’s Pediatric Heart Failure Toolkit. Download patient and care team friendly handouts below. Providers For more tools, including a Heart Failure Daily Communication Checklist and a Heart Failure Discharge Plan login
Pediatric Heart Failure Toolkit Read More »
ACTION defines and collects pediatric heart failure metrics to implement quality improvement initiatives. This study sought to describe medication use at the time of discharge for children admitted with acute
Establishing Baseline Metrics of Heart Failure Medication Use in Children Read More »
