Pediatric neuromuscular diseases encompass all disorders with onset in childhood and
where the primary area of pathology is in the peripheral nervous system. These conditions …

Duchenne muscular dystrophy (DMD) is one of the most devastating neuromuscular genetic
diseases caused by the absence of dystrophin. The continuous episodes of muscle …

Aims Epicardium and epicardium-derived cells are critical players in myocardial fibrosis.
Mesenchymal stem cell–derived extracellular vesicles (EVs) have been studied for cardiac …

Duchenne muscular dystrophy, like other muscular dystrophies, is a progressive disorder
hallmarked by muscle degeneration, inflammation, and fibrosis. Latent transforming growth …

X-linked myotubular myopathy (XLMTM) is a fatal neuromuscular disorder caused by loss of
function mutations in MTM1. At present, there are no directed therapies for XLMTM, and …

Genetic modifiers can serve as biomarkers for outcomes in DMD. Modifiers can alter
strength and ambulation in muscular dystrophy, and these same features can be used as …

In skeletal muscle, extracellular matrix (ECM) remodeling can either support the complete
regeneration of injured muscle or facilitate pathologic fibrosis and muscle degeneration …

The role of the transforming growth factor (TGF)-β pathway in myocardial fibrosis is well
recognized. However, the precise role of this signaling axis in cardiomyocyte (CM) biology is …

Mechanical ventilation (MV) is a life-saving measure for those incapable of adequately
ventilating or oxygenating without assistance. Unfortunately, even brief periods of MV result …

Genetic disruption of the dystrophin complex produces muscular dystrophy characterized by
a fragile muscle plasma membrane leading to excessive muscle degeneration. Two genetic …