Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

The first published description of therapeutic applications of antisense oligonucleotide
(ASO) technology occurred in the late 1970s and was followed by the founding of …

Nonischemic dilated cardiomyopathy (DCM) often has a genetic pathogenesis. Because of
the large number of genes and alleles attributed to DCM, comprehensive genetic testing …

Duchenne muscular dystrophy is an X-linked disease caused by the absence of functional
dystrophin in the muscle cells. Major advances have led to the development of gene …

Mutations in USH2A are among the most common causes of syndromic and non-syndromic
retinitis pigmentosa (RP). The two most recurrent mutations in USH2A, c. 2299delG and c …

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

Duchenne muscular dystrophy (DMD) is a rare, severe, progressive muscle-wasting disease
leading to disability and premature death. Patients lack the muscle membrane-stabilizing …

Antisense oligonucleotide (AON) therapies involve short strands of modified nucleotides that
target RNA in a sequence‐specific manner, inducing targeted protein knockdown or …

Despite significant advances in basic research, the treatment of degenerative diseases of
the nervous system remains one of the greatest challenges for translational medicine. The …

Highlights•Numerous pathophysiological features of DMD provide different therapeutic
avenues.•Pharmacolougical therapies target all muscles and are applicable to all DMD …