Exon Skipping Therapy

Exon skipping is a novel therapeutic approach to correct mutations in Duchenne muscular dystrophy (DMD) patients and restore dystrophin expression. To produce the dystrophin protein, 79 exons are needed. However, in DMD some exons are deleted (out-of-frame mutations) which causes a lack of functional dystrophin production. By skipping the nonfunctional exons (using an antisense oligonucleotide), a shortened dystrophin protein can be produced which may have some functionality. There are currently four approved exon skipping options (antisense oligonucleotides) for DMD – Amondys 45®, Exondys 51®, Vyondys 53®, and Viltepso®.

Additional Information

Exon skipping Information (basic)

Exon skipping Information (scientific/detailed)

Product Comparison

Brand Name	Generic	Indication	Cost (WAC)
Amondys 45	Casimersen	Treatment of Duchenne muscular dystrophy (DMD) in patients who have confirmed mutation of the DMD gene that is amenable to exon 45 skipping	$749,000 annual (30kg)
Exondys 51	Eteplirsen	Treatment of Duchenne muscular dystrophy (DMD) in patients who have confirmed mutation of the DMD gene that is amenable to exon 51 skipping	$749,000 annual (30kg)
Vyondys 53	Golodirsen	Treatment of Duchenne muscular dystrophy (DMD) in patients who have confirmed mutation of the DMD gene that is amenable to exon 53 skipping	$749,000 annual (30kg)
Viltepso	Viltolarsen	Treatment of Duchenne muscular dystrophy (DMD) in patients who have confirmed mutation of the DMD gene that is amenable to exon 53 skipping	$704,000 annual (30kg)