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.
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 »
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 »
Through the collection and use of real-world data, the field has gained greater understanding of best practices in care of these patients, as well as improved access to devices with pediatric labeling of devices previously approved only for adults. Recent and current work across the network’s major focus areas (VAD, heart failure, muscular dystrophy, and Fontan) are highlighted.
ACTION Oral Presentation Transplant Dystrophinopathy Transplantation in Males with Dystrophinopathy: An Advanced Cardiac Therapies Improving Outcomes Network (ACTION) Dystrophinopathy Registry Analysis Emily Hayes, MD et. al. Download abstract…
30th Annual Congress of World Muscle Society – ACTION Oral Presentation Read More »
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.
The Pulse — April 2025 Read More »
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.
The Pulse — February 2025 Read More »
Happy Fall to All! We had a successful meeting during the Single Ventricle Investigators Meeting in Denver this month, and it was great to see so many of you in person! Many new junior faculty and leaders of all experience levels have joined ACTION, and we are excited to continue our progress in the VAD, Heart Failure, Muscular Dystrophy, and Fontan spaces. We are grateful for the engagement and collaboration from all stakeholders that continue to excel our research, quality improvement, education, and advocacy efforts.
The Pulse — November 2024 Read More »
What: Poster Session When: Friday, October 11 from 2:15-3:15 pm Dystrophinopathy in ACTION; Analysis of the first 500 males enrolled in the Advanced Cardiac Therapies Improving Outcomes Network prospective
29th Annual Congress of World Muscle Society- ACTION Abstracts Poster Session Read More »
