… the only fully approved drugs used in DMD therapy able to slow disease progression. In the
… therapeutic approach aimed to restore dystrophin production and to preserve muscle mass, …

Genetic approaches for the diagnosis and treatment of inherited muscle diseases have … the
treatment of Duchenne muscular dystrophy (DMD), a muscle wasting disease where affected …

… dystrophin due to mutations in the dystrophin (DMD) gene [1,2]. Duchenne muscular dystrophy
is typically diagnosed … gait and whole-body skeletal muscle wasting, which progress due …

… of dystrophin Kobe we propose a novel way of gene therapy … This first in vitro evidence that
dystrophin pre-mRNA splicing … new therapeutic approach for Duchenne muscular dystrophy. …

… production of muscle dystrophin also prevent the production of two other full-length and,
depending on the location of the mutation, one or more shorter brain isoforms of dystrophin. It is …

… leading to development of cutting-edge therapies. … muscular dystrophy (DMD), Becker
muscular dystrophy (BMD) and limb girdle muscular dystrophies (LGMD), emphasising diagnosis …

… its effect on the production of dystrophin. Milder allelic forms of the disease also exist,
including intermediate muscular dystrophy and Becker muscular dystrophy, which cause loss of …

… of conditions that impair the function of muscles, motor neurons, peripheral … muscular
dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in the X-linked dystrophin …

Duchenne muscular dystrophy (DMD) is the commonest and best-known of the muscular
dystrophies. Being an X-linked disorder, it affects mainly boys. The disease gene was identified …

The term muscular dystrophy (MD) comprises various neuromuscular disorders that are
characterised by progressive muscle weakness affecting certain muscle groups, which are …