The Medical Letter on Drugs and Therapeutics
Deflazacort (Emflaza) for Duchenne Muscular Dystrophy
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Deflazacort (Emflaza – PTC Therapeutics), an oral corticosteroid, has been approved by the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients ≥5 years old. It has been available outside the US for many years. Deflazacort is the second drug to be approved for treatment of DMD; eteplirsen (Exondys 51), an antisense oligonucleotide approved for IV administration in patients with mutations of the dystrophin gene amenable to exon 51 skipping (about 13% of DMD cases), was the first.1

THE DISORDER — DMD is a progressive, X-linked, recessive, neuromuscular disorder characterized by decreased levels or complete absence of the protein dystrophin in muscle cells. Dystrophin is essential for maintenance of myocyte integrity. About 1 in 5000 infant males has DMD. Patients with DMD begin experiencing progressive muscle weakness and deterioration when they ... more      

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Title: Deflazacort (Emflaza) for Duchenne Muscular Dystrophy
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