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

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

Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disorder caused by loss of
dystrophin. Several therapeutic modalities are currently in clinical trials but none will achieve …

Duchenne muscular dystrophy (DMD) has been a major target for gene therapy
development for nearly 30 years. DMD is among the most common genetic diseases, and …

Duchenne muscular dystrophy is an inherited, progressive muscle-wasting disorder caused
by mutations in the dystrophin gene. An increasing variety of approaches are moving …

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 a severe muscle-wasting disorder for which there is
currently no effective treatment. This disorder is caused by mutations or deletions in the …

Despite the full cloning of the Dystrophin cDNA 35 years ago, no effective treatment exists
for the Duchenne Muscular Dystrophy (DMD) patients who have a mutation in this gene …

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 …

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 …