Most gene therapy approaches to genetic disorders aim to compensate loss-of-function by
introducing recombinant cDNA-based minigenes into diseased tissues. The current report …

A workshop entitled The Development of Antisense Oligonucleotide Therapies for
Duchenne Muscular Dystrophy, sponsored by the EU funded Clinical Network TREAT-NMD …

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

There are few disease-modifying therapeutics for neurodegenerative diseases, but
successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal …

Duchenne muscular dystrophy (DMD) is a lethal muscle disorder caused by mutations in the
DMD gene for which no mutation‐targeted therapy has been available thus far. However …

Oligonucleotide-mediated splicing modulation is a promising therapeutic approach for
Duchenne muscular dystrophy (DMD). Recently, eteplirsen, a phosphorodiamidate …

Dysregulation of RNA splicing causes many diseases and disorders. Several therapeutic
approaches have been developed to correct aberrant alternative splicing events for the …

Neuromuscular disorders such as Duchenne Muscular Dystrophy and Spinal Muscular
Atrophy are neurodegenerative genetic diseases characterized primarily by muscle …

Movement disorders are a group of neurological conditions characterized by abnormalities
of movement and posture. They are broadly divided into akinetic and hyperkinetic …

The two therapeutic approaches currently most advanced in clinical trials for Duchenne
muscular dystrophy are antisense‐mediated exon skipping and forced read‐through of …