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 …

Over the years various steroid trials have been conducted in Duchenne muscular dystrophy
(DMD). In children who are still able to walk as well as in those who are wheelchair‐bound …

The absence of dystrophin in Duchenne muscular dystrophy disrupts the dystrophin-
associated glycoprotein complex resulting in skeletal muscle fiber fragility and atrophy …

A therapeutic approach for Duchenne muscular dystrophy (DMD) is to up-regulate utrophin
in skeletal muscle in an effort to compensate for the lack of dystrophin. We previously …

Abstract X-linked adrenoleukodystrophy (X-ALD) is a severe neurodegenerative disease
caused by loss of function of the peroxisomal transporter ABCD1 (ALD), which results in …

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 …

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease arising from loss-
of-function mutations in the dystrophin gene and characterized by progressive muscle …

The muscular dystrophies and congenital myopathies are inherited diseases of the skeletal
muscle, which lead to a loss of muscle function and are often fatal. While many of the loci …

Aims Cardiac myopathies are the second leading cause of death in patients with Duchenne
and Becker muscular dystrophy, the two most common and severe forms of a disabling …

Duchenne muscular dystrophy (DMD) is a progressive, fatal, X-linked monogenic muscle
disorder caused by mutations in the DMD gene. In order to test treatments for DMD, a range …