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.
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.
Partnership of cardiologists with gene transfer therapy prescribers is essential to identifing patient-specific considerations that might influence risk for adverse cardiac events.
Purpose Cardiac disease results in significant morbidity and mortality in patients with muscular dystrophy (MD). Single centers have reported their ventricular assist device (VAD) experience in specific MDs and in
The results of an ACTION-wide survey of pediatric cardiology providers are detailed in the latest ACTION publication, Current Practices in Treating Cardiomyopathy and Heart Failure in Duchenne Muscular Dystrophy (DMD):
“The influence of mechanical circulatory support on post-transplant outcomes in pediatric patients” was published in the Journal of Heart and Lung Transplantation in June of 2021. The multicenter study from
The Influence of MCS on Post-Transplant Outcomes in Pediatric Patients Read More »
