The Medical Letter on Drugs and Therapeutics
Casimersen (Amondys 45) for Duchenne Muscular Dystrophy (online only)
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Casimersen (Amondys 45 – Sarepta), an IV antisense oligonucleotide, has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients who have mutations of the dystrophin gene that are amenable to exon 45 skipping (DMD-45), which occur in ~8% of DMD cases. Casimersen is the first drug to be approved for this indication and the fifth to be approved for treatment of DMD; the IV antisense oligonucleotides eteplirsen (Exondys 51), golodirsen (Vyondys 53), and viltolarsen (Viltepso) and the oral corticosteroid deflazacort (Emflaza) were approved earlier. The DMD gene consists of 79 exons; its long length makes it susceptible to mutations and deletions. ... more
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Title: Casimersen (Amondys 45) for Duchenne Muscular Dystrophy (online only)
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