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 …

Neuromuscular disorders encompass a heterogeneous group of conditions that impair the
function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being …

Objective To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression
in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon …

Background: Eteplirsen received accelerated FDA approval for treatment of Duchenne
muscular dystrophy (DMD) with mutations amenable to exon 51 skipping, based on …

Duchenne muscular dystrophy (DMD) is typically caused by mutations that disrupt the DMD
reading frame, but nonsense mutations in the 5′ part of the gene induce utilization of an …

Objective This study was undertaken to determine whether a low residual quantity of
dystrophin protein is associated with delayed clinical milestones in patients with DMD …

DMD pathogenic variants for Duchenne and Becker muscular dystrophy are detectable with
high sensitivity by standard clinical exome analyses of genomic DNA. However, up to 7% of …

Becker muscular dystrophy is caused by mutations in the DMD gene that permit significant
residual dystrophin protein expression in patient muscle. This is in contrast to DMD gene …

The interactions between nutrition and metabolism and skeletal muscle have long been
known. Muscle is the major metabolic organ—it consumes more calories than other organs …

Duchenne muscular dystrophy (DMD) is a degenerative muscular disease affecting roughly
one in 5000 males at birth. The disease is often caused by inherited X-linked recessive …