Muscular Dystrophy

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 »

Improving Pediatric Heart Failure Outcomes Through Collaboration and Innovation: The Current Status of the Advanced Cardiac Therapies Improving Outcomes Network (ACTION)

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.

Improving Pediatric Heart Failure Outcomes Through Collaboration and Innovation: The Current Status of the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) 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.

The Pulse — April 2025 Read More »

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.

Cardiac treatment for Duchenne muscular dystrophy: consensus recommendations from the ACTION muscular dystrophy committee Read More »

The Pulse eNewsletter - February 2025

The Pulse — February 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.

The Pulse — February 2025 Read More »

Taking ACTION to detect myocarditis related to recombinant gene transfer therapy for Duchenne Muscular Dystrophy; Consensus recommendations for cardiac surveillance

Partnership of cardiologists with gene transfer therapy prescribers is essential to identifing patient-specific considerations that might influence risk for adverse cardiac events.

Taking ACTION to detect myocarditis related to recombinant gene transfer therapy for Duchenne Muscular Dystrophy; Consensus recommendations for cardiac surveillance Read More »

The Pulse eNewsletter July 2024

The Pulse — July 2024

Dear Community, 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, and resources. Special thanks to the Muscular Dystrophy & Heart Failure committees for new harmonization protocols, and to the Fontan Referral Improvement Project leadership & committee for launching the Provider Grand Rounds deck for Advanced Therapeutic Options for Fontan Patients!

The Pulse — July 2024 Read More »