The Medical Letter on Drugs and Therapeutics
Eteplirsen (Exondys 51) for Duchenne Muscular Dystrophy
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Eteplirsen (Exondys 51 – Sarepta), an antisense oligonucleotide, has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients who have a mutation of the dystrophin gene that is amenable to exon 51 skipping. It is the first drug to be approved for treatment of DMD.

THE DISORDER — DMD is a progressive, X-linked, recessive, neuromuscular disorder characterized by a near-complete absence of the protein dystrophin in muscle cells. Dystrophin is essential for maintenance of myocyte integrity.1 About 1 in 3600 infant males has DMD. Patients with DMD begin experiencing progressive muscle weakness and deterioration when they are 3-5 years old and usually become nonambulatory by their early teenage years. Death almost always occurs before age 40. High-dose prednisone therapy (0.75 mg/kg/day) is commonly used to slow ... more      

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Title: Eteplirsen (Exondys 51) for Duchenne Muscular Dystrophy
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