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 »
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 »
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.
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 »
