Dystrophin deficiency results in lethal Duchenne muscular dystrophy (DMD). Substituting
missing dystrophin with abbreviated microdystrophin has dramatically alleviated disease in …

Although there is good experimental data that utrophin, the autosomal analog of dystrophin,
can ameliorate the phenotype in dystrophinopathies, there is scant evidence from human …

A possible treatment for Duchenne muscular dystrophies would be to compensate for
dystrophin loss by increasing the expression of utrophin, another cytoskeletal protein of the …

Duchenne muscular dystrophy results from the absence of dystrophin, a cytoskeletal protein.
Previously, we have shown in a transgenic mouse model of the disease (mdx) that high …

A promising therapeutic approach for Duchenne muscular dystrophy (DMD) is exon skipping
using antisense oligonucleotides (AOs). In-frame deletions of the hinge 3 region of the …

Purpose SMT C1100 is a utrophin modulator being evaluated as a treatment for Duchenne
muscular dystrophy (DMD). This study, the first in pediatric DMD patients, reports the safety …

Duchenne muscular dystrophy is a severe X-linked inherited muscle wasting disorder
caused by mutations in the dystrophin gene. Adeno-associated virus (AAV) vectors have …

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder caused by
the lack of a subsarcolemmal protein, dystrophin. We have previously shown that the …

Duchenne muscular dystrophy (DMD) is a progressive X-linked degenerative muscle
disease due to mutations in the DMD gene. Genetic confirmation has become standard in …

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 …