Department of Defense Funds ACTION to Advance Duchenne Muscular Dystrophy Cardiac Research
ACTION receives $910,000 Department of Defense award to study the evolving impact of therapies on heart disease in Duchenne muscular dystrophy
Duchenne Muscular Dystrophy
Use of SGLT2 Inhibitors in Dystrophin Deficient Cardiomyopathy: A Multi-center Study Using the ACTION Registry
A new multicenter study suggests SGLT2 inhibitors — a diabetes drug class — may offer a well-tolerated way to modestly improve heart function in kids with dystrophin-deficient cardiomyopathy.
Meeting Report: Expanding Access to Advanced Cardiac Therapies, Including Ventricular Assist Devices (VADs) and Heart Transplantation in Muscular Dystrophy
Following a 2024 expert convening, a comprehensive clinical protocol was created to support VAD and transplant candidacy in Duchenne muscular dystrophy, translating consensus into real-world implementation.
VAD and Heart Transplantation in DMD: Guidance for Providers
Check out the newest Harmonized Protocol for Providers caring for VAD and Heart Transplantation patients with Duchenne Muscular Dystrophy.
VAD and Heart Transplantation in DMD: Guidance for Providers Read More »
Dystrophinopathy in ACTION: The first 500 males enrolled in the Advanced Cardiac Therapies Improving Outcomes Network prospective dystrophinopathy registry
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.
The Pulse — Nov 2025
Happy Fall to All! We have many exciting updates to share, such as new pediatric Impella education materials for Providers and Patients/Families, a debrief of our incredibly successful first VAD Bootcamp, and many new publications and other tools. As we move into the holiday season, we reflect on our continued appreciation and thankfulness for the engagement and collaboration from all stakeholders that continue to excel our research, quality improvement, education, and advocacy efforts.
The Pulse — Nov 2025 Read More »
The Pulse — Aug 2025
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 »
Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy
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 »
The Pulse — April 2025
Let’s make 2025 our best year yet! We wrapped 2024 with the exciting announcement for the FDA expanded label of Johnson & Johnson MedTech’s Impella Support System to include treatment for certain children suffering from heart failure. Achievements like this would not be possible without the continued efforts and dedication of our ACTION centers providing care to these patients, as well as entering, cleaning, and adjudicating the data. We also continued access to the Berlin Heart Active Driver under the extended trial, and we published many new ACTION manuscripts.
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Cardiac treatment for Duchenne muscular dystrophy: consensus recommendations from the ACTION muscular dystrophy committee
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.
