Duchenne muscular dystrophy (DMD) is a devastating disease featuring skeletal muscle
wasting, respiratory insufficiency, and cardiomyopathy. Historically, respiratory failure has …

Duchenne muscular dystrophy (DMD), a fatal X-linked recessive disorder, is caused mostly
by frame-disrupting, out-of-frame deletions in the dystrophin (DMD) gene. Antisense …

Duchenne muscular dystrophy (DMD), a severe hereditary disease affecting 1 in 3,500 boys,
mainly results from the deletion of exon (s), leading to a reading frameshift of the DMD gene …

Clinical implementation of two recently approved antisense RNA therapeutics–Exondys51®
to treat Duchenne muscular dystrophy (Duchenne MD) and Spinraza® as a treatment for …

Rare diseases affect almost 500 million people globally, predominantly impacting children
and often leading to significantly impaired quality of life and high treatment costs. While …

Dystrophinopathies are X-linked recessive muscle disorders caused by mutations in the
dystrophin (DMD) gene that include deletions, duplications, and point mutations. Correct …

Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disorder generally caused by
out-of-frame mutations in the DMD gene. In contrast, in-frame mutations usually give rise to …

Mutations of the dystrophin DMD gene, essentially deletions of one or several exons, are the
cause of two devastating and to date incurable diseases, Duchenne (DMD) and Becker …

Duchenne muscular dystrophy (DMD) is a devastating disease that results in life-limiting
complications such as loss of skeletal muscle function as well as respiratory and cardiac …

Exon skipping therapy has recently received attention for its ability to convert the phenotype
of lethal Duchenne muscular dystrophy (DMD) to a more benign form, Becker muscular …