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FDA Approves Exondys 51 (eteplirsen) for Duchenne Muscular Dystrophy

Posted 21 Sep 2016 by Drugs.com

September 19, 2016 – The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD. “Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eage ... Read more

Related support groups: Muscular Dystrophy, Duchenne Muscular Dystrophy, Exondys 51, Eteplirsen

FDA Approves Exondys 51 - First Drug to Treat Duchenne Muscular Dystrophy

Posted 20 Sep 2016 by Drugs.com

MONDAY, Sept. 19, 2016 – The U.S. Food and Drug Administration on Monday granted preliminary approval to the first drug for a rare form of muscular dystrophy. Exondys 51 (eteplirsen) was granted accelerated approval to treat Duchenne muscular dystrophy, a genetic disorder that progressively weakens the muscular systems of its victims. Most are in a wheelchair by their teens and do not survive past their 20s or 30s. "Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease," Dr. Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, said in an agency news release. "In rare diseases, new drug development is especially challenging, due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders," Woodcock said. "Accelerated ... Read more

Related support groups: Muscular Dystrophy, Duchenne Muscular Dystrophy, Exondys 51, Eteplirsen

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Duchenne Muscular Dystrophy

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Exondys 51

Eteplirsen Patient Information at Drugs.com