Treatment For Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is an inherited disorder that results in progressive muscle weakness and loss of muscle mass, primarily affecting males. Duchenne muscular dystrophy results from non-sense or frame-shifting variant(s) in the Duchenne muscular dystrophy gene which is responsible for producing dystrophin, a cohesive protein essential for maintaining muscle support and strength. Antisense oligonucleotides are short, synthetic, single-stranded oligodeoxynucleotides that selectively bind to specific exons of the dystrophin pre-messenger RNA causing the exon to be skipped and thereby repairing the mutated reading frame resulting in production of an internally truncated, yet functional, dystrophin protein. Four antisense oligonucleotides—eteplirsen, golodirsen, viltolarsen, and casimersen have been approved by the U.S. Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy. Each targets a specific exon. For example, eteplirsen targets skipping of exon 51, golodirsen and viltolarsen target skipping of exon 53, and casimersen targets skipping of exon 45.