Gene therapy for Duchenne muscular dystrophy : Current Opinion in Neurology Gene therapy
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Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder
that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the …

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 the most common form of muscular dystrophy in
childhood. Mutations of the DMD gene destabilize the dystrophin associated glycoprotein …

Background Duchenne muscular dystrophy (DMD) is a severe rare progressive inherited
neuromuscular disorder, leading to loss of ambulation (LOA) and premature mortality. The …

Duchenne muscular dystrophy (DMD) is an X-linked progressive degenerative muscle
disorder caused by the absence of dystrophin. There is no curative therapy, although …

Duchenne muscular dystrophy (DMD) is a severe, progressive disease that affects 1 in 3600–
6000 live male births. Although guidelines are available for various aspects of DMD …

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 …

Duchenne muscular dystrophy (DMD) is a devastating, rare disease. While clinically
described in the 19 th century, the genetic foundation of DMD was not discovered until more …

Duchenne muscular dystrophy (DMD) is an X-linked inherited neuromuscular disorder due
to mutations in the dystrophin gene. It is characterized by progressive muscle weakness and …