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Duchenne Muscular Dystrophy News

Scientists Turn Skin Cells Into Muscle Cells, a Potential Boon for Research

Posted 9 Jan 2018 by

TUESDAY, Jan. 9, 2018 – In a potential advance for medical research, scientists say they've created the first functioning human muscle from skin cells. The breakthrough could lead to better genetic or cell-based therapies, as well as furthering investigations into the causes and treatment of muscular disorders, the Duke University team said. "The prospect of studying rare diseases is especially ...

FDA Approves Emflaza (deflazacort) to treat Duchenne Muscular Dystrophy

Posted 10 Feb 2017 by

February 9, 2017 – The U.S. Food and Drug Administration today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system. ...

Emflaza Approved for Duchenne Muscular Dystrophy

Posted 9 Feb 2017 by

THURSDAY, Feb. 9, 2017 --Emflaza (deflazacort) has been approved by the U.S. Food and Drug Administration to treat Duchenne muscular dystrophy in people five years and older, the agency said Thursday in a news release. The corticosteroid is designed to reduce inflammation and suppress the immune system in people with DMD, the most common type of muscular dystrophy. The FDA said it's the first ...

US Families Spend 1.5 Billion Hours Yearly on Kids With Special Health Needs

Posted 27 Dec 2016 by

TUESDAY, Dec. 27, 2016 – Families in the United States spend 1.5 billion hours each year providing home health care to their chronically ill or disabled children, a new study shows. The hours of health care these parents and other family members provide would cost $36 billion if performed by home health workers who received the going rate – or $12 billion if unskilled workers receiving minimum ...

FDA Approves Exondys 51 (eteplirsen) for Duchenne Muscular Dystrophy

Posted 21 Sep 2016 by

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 ...

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

Posted 20 Sep 2016 by

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 ...

Exondys 51 First Drug Approved for Duchenne Muscular Dystrophy

Posted 19 Sep 2016 by

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 ...

Gene Therapy in Dogs Shows Promise for Muscular Dystrophy

Posted 2 Nov 2015 by

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 ...

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Further Information

Related Condition Support Groups

Muscular Dystrophy

Related Drug Support Groups

Exondys 51, eteplirsen, deflazacort, Emflaza