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Duchenne Muscular Dystrophy News

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

First Drug Approved for Duchenne Muscular Dystrophy

Posted 19 Sep 2016 by Drugs.com

MONDAY, Sept. 19, 2016 – Exondys 51 (eteplirsen) injection has been approved by the U.S. Food and Drug Administration for certain people with Duchenne muscular dystrophy (DMD). It's the first drug to gain FDA approval for the condition. Exondys 51 is approved for people with a specific mutation of the dystrophin gene, affecting some 13 percent of people with the disorder. DMD, the most common form of muscular dystrophy, is a rare genetic condition that leads to ongoing muscular deterioration and weakness. Symptoms typically first appear between ages three and five, mostly among boys without a family history of the disorder, the FDA said Monday in a news release. People with DMD typically die in their 20s or 30s from the progressive disorder, which affects about one of every 3,600 males worldwide. Accelerated approval was granted to the new drug based on clinical studies that found an ... Read more

Related support groups: Muscular Dystrophy, Duchenne Muscular Dystrophy

Gene Therapy in Dogs Shows Promise for Muscular Dystrophy

Posted 2 Nov 2015 by Drugs.com

MONDAY, Nov. 2, 2015 – Using gene therapy, researchers report they've successfully treated muscular dystrophy in dogs. They believe this could pave the way for clinical trials of the treatment in humans within the next few years. The dogs had Duchenne muscular dystrophy, which is the most common form of the disease in humans and primarily affects boys. Patients lose their ability to walk and breathe as they get older, the researchers said. "This is the most common muscle disease in boys, and there is currently no effective therapy," said study author Dongsheng Duan, a professor of medical research at the University of Missouri School of Medicine. "This discovery took our research team more than 10 years, but we believe we are on the cusp of having a treatment for the disease," he added in a university news release. However, results of animal studies often fail to be replicated in human ... Read more

Related support groups: Muscular Dystrophy, Duchenne Muscular Dystrophy

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Exondys 51, eteplirsen