Exon Skipping Dominates DMD Treatment, But What’s on Horizon?

Exon Skipping Dominates DMD Treatment, But What’s on Horizon?

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Exon Skipping Dominates DMD Treatment, But What’s on Horizon?

With the recent approval of Amondys 45 (casimersen) by Sarepta Therapeutics, there are now four exon-skipping therapies available in the U.S. for eligible patients with Duchenne muscular dystrophy (DMD). The others are Exondys 51 (eteplirsen) and Vyondys 53 (golodirsen), also by Sarepta, and Nippon Shinyaku’s Viltepso (viltolarsen) — all approved in the past five years and, combined, able to treat nearly 30% of all DMD cases. But what makes these therapies special, and how well do they benefit these…

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