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

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

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 university news release. Poor lip control caused by facial paralysis can cause drooling, spillage of food and drink, and difficulty making sounds that require fully closing the lips, such as the letters "b" and "p." Many of these people are also self-conscious about their appearance, Boahene said. He and his colleagues worked with 22 people who had facial paralysis on one side of the mouth and three people with muscular dystrophy who had lost control of both sides of the mouth. The patients were given ... Read more

Related support groups: Ischemic Stroke, Muscular Dystrophy, Cerebral Palsy, Intracranial Hemorrhage, Paralytic Disorder, Facial Lipoatrophy

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 whites or blacks. "There were always some rather crude estimates of how common these muscular dystrophies are," study corresponding author Paul Romitti, an epidemiologist at the University of Iowa, said in a university news release. "It tells us that they're still an important public health concern." In the study, the team analyzed data from children born in six states – Arizona, Colorado, Georgia, Hawaii, Iowa and New York – between 1982 and 2011. The reasons why the disorders affect ... Read more

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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 were randomly selected to join other patients in seeing a doctor or to have a one-on-one meeting with a doctor. The group visits included five to eight patients with the same disorder who saw a neurologist for 90 minutes to two hours. The individual appointments lasted 20 to 30 minutes. The patients then reported on their quality of life one week, three months and six months after their appointments. On average, patients who attended the group visits scored three points higher on ... Read more

Related support groups: Muscular Dystrophy

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 dystrophy have a blood flow abnormality – delivery of blood and oxygen to their muscles – that does not increase the way it should during mild exercise," said lead researcher Dr. Ronald Victor, the associate director of the Cedars-Sinai Heart Institute in Los Angeles. Because the blood vessels do not enlarge in a normal way in Duchenne muscular dystrophy, muscles are starved for oxygen, according to Victor. Cialis and Viagra work by enlarging blood vessels, thus increasing blood flow, which is ... Read more

Related support groups: Viagra, Cialis, Muscular Dystrophy, Sildenafil, Tadalafil, Revatio, Adcirca

Docs: Heart Device Might Be Breakthrough for Muscular Dystrophy

Posted 17 Oct 2012 by Drugs.com

WEDNESDAY, Oct. 17 – A man with Duchenne muscular dystrophy who received a device to help his heart's left ventricle pump blood throughout his body could represent a breakthrough in the treatment of the disease, according to his doctors. Jason Williams, 29, of Peebles, Ohio, is believed to be one of the first U.S. patients with Duchenne muscular dystrophy to receive an implanted ventricular assist device, the Cincinnati Children's Hospital Medical Center team said. Ventricular assist devices are mechanical pumps implanted in the chest to help a weakened heart pump blood to the rest of the body. Eighty percent of boys and men with Duchenne muscular dystrophy die of heart failure, according to a medical center news release. Due to the severity of their muscle disease, they are not typically candidates for heart transplant or some other treatment options available to patients with other ... Read more

Related support groups: Muscular Dystrophy

Type of Muscular Dystrophy Linked to Raised Cancer Risk

Posted 13 Dec 2011 by Drugs.com

TUESDAY, Dec. 13 – People with a certain type of muscular dystrophy have twice the risk of being diagnosed with cancer as the general population, although the actual risk is relatively low, a new study finds. Myotonic muscular dystrophy (MMD) is characterized by progressive weakness and muscular wasting that can affect many muscles, including those of the face, neck, hands and feet. The condition can range from mild to severe. The study, published in the Dec. 14 issue of the Journal of the American Medical Association, included 1,658 patients in Denmark and Sweden who were diagnosed with MMD between 1977 and 2008, and followed until they were diagnosed with cancer, died or emigrated. During follow-up, about 40 percent of the patients died and about 6 percent developed cancer. The number of MMD patients who developed cancer (104) was twice as high as the 52 cases that would be expected ... Read more

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Myotonia Congenita, Duchenne Muscular Dystrophy, Neurologic Disorder