Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

Abstract The Dystrophin Glycoprotein Complex (DGC) is a large multi-protein complex that
links cytoskeleton actin to the extracellular matrix. This complex is critical in maintaining the …

Duchenne muscular dystrophy (DMD) is a severe progressive muscular disorder caused by
reading frame disrupting mutations in the DMD gene, preventing the synthesis of functional …

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by the
lack of functional dystrophin. There is no cure, but several clinical trials aimed to restore the …

Various therapeutic approaches have been studied for the treatment of Duchenne muscular
dystrophy (DMD), but none of these approaches have led to significant long-term effects in …

Gene mutations causing loss of dystrophin result in the severe muscle disease known as
Duchenne muscular dystrophy (DMD). Despite efforts at genetic repair, DMD therapy …

Objective Sporadic inclusion body myositis (sIBM), an intractable progressive muscle
disease, frequently occurs in older persons. sIBM pathogenesis may involve protein …

Duchenne muscular dystrophy (DMD) is an X‐linked myopathy caused by dystrophin
deficiency. Dystrophin is present intracellularly at the sarcolemma, connecting actin to the …

Background Analysis of muscle biopsies allowed to characterize the pathophysiological
changes of Duchenne and Becker muscular dystrophies (D/BMD) leading to the clinical …

In the last two decades, antisense oligonucleotides (AONs) that induce corrective exon
skipping have matured as promising therapies aimed at tackling the dystrophin deficiency …