Neuromuscular disorders encompass a heterogeneous group of conditions that impair the
function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being …

Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

Duchenne muscular dystrophy (DMD) is caused by mutations in the gene that encodes the
427‐kDa cytoskeletal protein dystrophin. Increased knowledge of the function of dystrophin …

Duchene Muscular Dystrophy (DMD) is the most frequent muscular dystrophy and one of the
most severe due to the absence of the dystrophin protein. Typical pathological features …

Dystrophinopathies are diseases caused by mutations in the Duchenne Muscular Dystrophy
gene (DMD) encoding the dystrophin protein. Depending on the type of mutation, patients …

Although the molecular defect causing Duchenne/Becker muscular dystrophy (DMD/BMD)
was identified nearly 20 years ago, the development of effective therapeutic strategies has …

New Findings What is the topic of this review? This review highlights recent progress in
genetically based therapies targeting the primary defect of Duchenne muscular dystrophy …

Muscular dystrophies primarily affect skeletal muscle. Mutations in a large number of genes,
mainly encoding cytoskeletal proteins, cause different forms of dystrophy that compromise …

Despite an expansive wealth of research following the discovery of the DMD gene 25 years
ago, there is still no curative treatment for Duchenne muscular dystrophy. However, there …