A retooled drug that restores ionic balance and cardiac function in dystrophin deficient hearts

Duchenne muscular dystrophy (DMD) is a devastating and fatal disease affecting ~1 in 3500 boys with dystrophin gene mutations [1,2]. Dystrophin plays important roles in sarcolemma stability and cell signaling in skeletal, smooth, and cardiac muscles [1,2]. DMD patients often die at a young age from dilated cardiomyopathy (DCM) or respiratory dysfunction. The use of ventilators has reduced respiratory failure in DMD patients and DCM associated heart failure (HF) is now a major cause of DMD patient death [1,2].

Source: International Journal of Cardiology - June 2, 2020 Category: Cardiology Authors: Ryan D. Wuebbles, Dean J. Burkin Source Type: research