Diseases News & Updates

Exondys 51 (eteplirsen) First drug for Duchenne muscular dystrophy

Exondys 51

The U.S. Food and Drug Administration has approved Exondys 51 (eteplirsen) injection, the first of its type to treat patients suffering from Duchenne muscular dystrophy due to mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.

Duchenne muscular dystrophy is a genetic disorder characterized by progressive muscle deterioration and weakness caused by an absence of dystrophin, a protein that helps to keep muscle cells intact. The first symptoms are usually seen between three and five years of age, and worsen over time. The disease often occurs in people without a known family history mainly affects boys, but in rare cases it can affect girls. DMD occurs in about one out of every 3,600 male infants worldwide.

The complete press release from FDA :


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