The X-linked muscle-wasting disease Duchenne muscular dystrophy is caused by mutations
in the gene encoding dystrophin. There is currently no effective treatment for the disease; …

Current molecular genomic approaches to human genetic disorders have led to an
explosion in the identification of the genes and their encoded proteins responsible for these …

Skeletal muscle continuously adapts to changes in its mechanical environment through
modifications in gene expression and protein stability that affect its physiological function …

Almost every muscle contains muscle spindles. These delicate sensory receptors inform the
central nervous system (CNS) about changes in the length of individual muscles and the …

Duchenne muscular dystrophy (DMD) is a lethal, progressive muscle wasting disease
caused by a loss of sarcolemmal bound dystrophin, which results in the death of the muscle …

Dystrophin is a cytoskeletal protein of muscle fibers; its loss in humans leads to Duchenne
muscular dystrophy, an inevitably fatal wasting of skeletal and cardiac muscle. mdx mice …

Highlights•Numerous pathophysiological features of DMD provide different therapeutic
avenues.•Pharmacolougical therapies target all muscles and are applicable to all DMD …

The immune response to acute muscle damage is important for normal repair. However, in
chronic diseases such as many muscular dystrophies, the immune response can amplify …

Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …

Autosomal recessive polycystic kidney disease (ARPKD) is an infantile form of PKD
characterized by fusiform dilation of collecting ducts and congenital hepatic fibrosis. The …