Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …

Objective To describe the quantification of novel dystrophin production in patients with
Duchenne muscular dystrophy (DMD) after long-term treatment with eteplirsen. Methods …

Importance An unmet need remains for safe and efficacious treatments for Duchenne
muscular dystrophy (DMD). To date, there are limited agents available that address the …

A large and complex gene on the X chromosome encodes dystrophin. Many mutations have
been described in this gene, most of which affect the expression of the muscle isoform, the …

Antisense‐mediated exon skipping aiming for reading frame restoration is currently a
promising therapeutic application for Duchenne muscular dystrophy (DMD). This approach …

The severe Duchenne and milder Becker muscular dystrophy are both caused by mutations
in the DMD gene. This gene codes for dystrophin, a protein important for maintaining the …

Becker muscular dystrophy (BMD) is a variant of dystrophin deficiency resulting from DMD
gene mutations. Phenotype is variable with loss of ambulation in late teenage or late mid-life …

Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy are caused by
mutations in the dystrophin-encoding DMD gene. Large deletions and duplications are most …