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Muscular Dystrophy News

Related terms: Myotonic Muscular Dystrophy, Myotonic Dystrophy, Dystrophia Myotonica, Steinert's Disease, Becker Muscular Dystrophy, MD, Emery-Dreifuss Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy

Gene 'Editing' in Dog Study Shows Promise for Kids With Muscular Dystrophy

Posted 30 Aug 2018 by Drugs.com

THURSDAY, Aug. 30, 2018 – For the first time, scientists report using gene-editing technology to halt the progression of muscular dystrophy in dogs – suggesting a possible breakthrough for children with a form of the disease. Reporting in the current issue of Science, researchers describe how they used CRISPR technology to edit a naturally occurring genetic flaw that causes a version of ...

FDA Approves Emflaza (deflazacort) to treat Duchenne Muscular Dystrophy

Posted 10 Feb 2017 by Drugs.com

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. ...

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

Posted 27 Dec 2016 by Drugs.com

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 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 ...

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 ...

Exondys 51 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 ...

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 ...

Lip Injections May Ease Challenges of Facial Paralysis

Posted 18 Jun 2015 by Drugs.com

THURSDAY, June 18, 2015 – People with facial paralysis may benefit from cosmetic lip surgery, a preliminary study finds. Facial paralysis is "a very large problem that can occur because of stroke, Bell's palsy, muscular dystrophy, trauma and birth defects," Dr. Kofi Boahene, a facial plastic and reconstructive surgeon at Johns Hopkins University School of Medicine in Baltimore, said in a ...

Researchers Measure Scope of Muscular Dystrophies That Strike Boys

Posted 20 Feb 2015 by Drugs.com

FRIDAY, Feb. 20, 2015 – A new study estimates the prevalence of two types of muscular dystrophy that strike mostly boys. A team led by researchers from the University of Iowa found that about one in 5,000 boys between the ages of 5 and 9 have either Duchenne muscular dystrophy or Becker muscular dystrophy. The researchers also found that Hispanic boys were struck by the disorders more often than ...

Group Doctor Visits May Benefit Muscle Disorder Patients

Posted 18 Jun 2014 by Drugs.com

WEDNESDAY, June 18, 2014 – People with neuromuscular disorders may gain more benefit from group doctor visits than individual appointments, Dutch researchers say. Examples of these types of disorders include amyotrophic lateral sclerosis (Lou Gehrig's disease) and muscular dystrophy. The new study included 272 patients who were due for their annual neurologist appointment. The study participants ...

Viagra, Cialis May Help Duchenne Muscular Dystrophy Patients

Posted 7 May 2014 by Drugs.com

WEDNESDAY, May 7, 2014 – Drugs normally prescribed to treat erectile dysfunction in adult males may help boys who have a muscle disease called Duchenne muscular dystrophy, according to a new study. In the small study including just 10 boys with the disease, researchers found that the popular drugs Viagra and Cialis improved blood flow to the boys' weakened muscles. "Boys with Duchenne muscular ...

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Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Myotonia Congenita, Neurologic Disorder