Rate of Change in Cardiac Magnetic Resonance Imaging Measures Is Associated With Death in Duchenne Muscular Dystrophy.
Joseph R StarnesMeng XuKristen George-DurrettKimberly CrumFrank J RaucciChristopher F SpurneyKan N HorLinda H CripeNazia HusainSujatha BuddheKatheryn GambettaJaclyn G TamaroffJames C SlaughterLarry W MarkhamJonathan H SoslowPublished in: Journal of the American Heart Association (2024)
Duchenne muscular dystrophy death is associated with the rate of change in left ventricular ejection fraction, midcircumferential strain, and ventricular volumes. Aggressive medical therapy to decrease the rate of progression may improve the mortality rate in this population. A decrease in the rate of progression may serve as a valid surrogate outcome for therapeutic trials.