Beth Kaufman

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.

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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.

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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 »

Initial Multicenter Experience with Ventricular Assist Devices in Children and Young Adults with Muscular Dystrophy: An ACTION Registry Analysis

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

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