Introduction Exon skipping compounds restoring the dystrophin transcript reading frame
have received regulatory approval for Duchenne muscular dystrophy (DMD). Recently, focus …

Introduction: Exon skipping is a therapeutic approach for Duchenne muscular dystrophy
(DMD) that has been in development for close to two decades. This approach uses …

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 …

Exondys 51 is the first therapy for Duchenne muscular dystrophy (DMD) to have been
granted accelerated approval by the FDA. Approval was granted based on using dystrophin …

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 rare, severe, progressive muscle-wasting disease
leading to disability and premature death. Patients lack the muscle membrane-stabilizing …

Molecular treatments for Duchenne muscular dystrophy (DMD) are already in clinical
practice. One particular means is exon skipping, an approach which has more than 15 years …

Duchenne muscular dystrophy (DMD) is a progressive muscle disease caused by loss of
dystrophin protein, encoded by the DMD gene. DMD manifests early in childhood as …

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 …

Introduction: Duchenne muscular dystrophy (DMD), one of the most common and lethal
genetic disorders, is caused by mutations of the dystrophin gene. Removal of an exon or of …