Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive
muscle weakness, is caused by the absence or a decreased amount of the muscle …

These novel therapies that act to correct the primary genetic defect of dystrophin deficiency
are among the first generation of therapies tailored to correct specific mutations in humans …

Duchene Muscular Dystrophy (DMD) is the most frequent muscular dystrophy and one of the
most severe due to the absence of the dystrophin protein. Typical pathological features …

Duchenne muscular dystrophy (DMD) is an X-linked hereditary disease characterized by
progressive muscle wasting due to modifications in the DMD gene (exon deletions …

Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

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

Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Muscular dystrophies primarily affect skeletal muscle. Mutations in a large number of genes,
mainly encoding cytoskeletal proteins, cause different forms of dystrophy that compromise …

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 …