Category Archives: Muscular Dystrophy Treatment

AMBITION FOR MD Ambition for Defying Muscular Dystrophy. By: DefyingDystrophy … Continue reading →

Muscular Dystrophy Treatment and Symptoms XXXXXX◅ Muscular Dystrophy treatment mdash; Finding the right information about Muscular Dystrophy treatment symptoms, is crucial to managing Muscular Dystroph... By: MainMDcom … Continue reading →

Duchenne Muscular Dystrophy and Becker's Muscular Dystrophy (DMD BMD) A pathology presentation made voluntarily for the sake of fun of video making by a 1st year medical student. Images and short clips are taken from various ot... By: Denise Zhang … Continue reading →

There will be a benefit for a Berwick woman diagnosed with Limb Girdle Muscular Dystrophy(LGMD)Saturday, June 15. The benefit will help assist in getting her a wheelchair accessible van. There will be food, bake sale, raffle, auction, etc. The organizers are in need of any type of donations: items for the raffle, musicians to perform, items to auction, sweets for the bake sale, bottled water, canned soda, and/or monetary donations. The organizers have some items to auction already including signed sports items by football stars, flat screen TV, his & her bikes. Click here to view the benefit on Facebook. Jennifer DeHart Sons is a single mom and is wheelchair bound for the most part. She can do things for herself as long as it can be done from her chair. Jennifer needs help with necessities in life that most people take for granted. She needs help with getting in and out of her chair, in and out of bed, on and off the toilet, in and out the car. Her senior year in high school she was an active regular teenager. Routine blood work showed abnormalities. The doctors diagnosed her in 1994 with a muscle disease called Polymyositis, which … Continue reading →

NEW YORK (AP) -- Shares of Sarepta Therapeutics Inc. fell Thursday after the drug developer forecast a larger-than-expected loss in 2013. Sarepta has no approved products and is studying a drug called eteplirsen as a treatment for Duchenne muscular dystrophy, a rare genetic disease that causes increasing muscle weakness. It is also researching a treatment for the deadly Marburg virus under an agreement with the U.S. Department of Defense. The Cambridge, Mass., company the company took a bigger loss in the fourth quarter because of a charge related to the increasing value of its stock. Sarepta lost $62.1 million, or $2.36 per share, in the latest quarter. That included a $51.8 million charge related to stock warrants. The company reported a loss of $1.4 million, or 6 cents per share, in the year-ago quarter. Revenue fell to $7.3 million from $13.6 million Analysts were expecting a loss of 27 cents per share and $7.1 million in revenue, according to FactSet. Companies often take charges when their stock rapidly increases in value to reflect greater liabilities. Sarepta's stock price rose more than fourfold in 2012 as it reported positive trial data for eteplirsen. The shares finished the year at $23.79 after … Continue reading →

Mar. 7, 2013 Researchers at Johns Hopkins have established a high-efficiency cell-cell fusion system, providing a new model to study how fusion works. The scientists showed that fusion between two cells is not equal and mutual as some assumed, but, rather, is initiated and driven by one of the fusion partners. The discovery, they say, could lead to improved treatments for muscular dystrophy, since muscle regeneration relies on cell fusion to make muscle fibers that contain hundreds or even thousands of nuclei. The study reveals two critical components that have to be present for cell fusion to happen, explains Elizabeth Chen, Ph.D., an associate professor of molecular biology and genetics in the Johns Hopkins University Institute for Basic Biomedical Sciences. Intriguingly, she says, one of these vital components actually changes the structure of one cell's scaffolding -- its cytoskeleton -- to form protrusions that push their way into the other cell to initiate fusion. Chen's research group had seen this before; using very high-resolution electron microscopy, they showed in 2010 that in developing fly muscles, a muscle cell merges with another muscle cell by extending finger-like protrusions into its fusion partner. But cell fusion is not only behind muscle building, … Continue reading →

P/>BOSTON (TheStreet) -- A GlaxoSmithKline (GSK) scientist, Dr. Rohit Batta, may have been trying to soothe the nerves of Duchenne muscular dystrophy parents when he provided another update about drisapersen on Monday. I believe he raised even more concerns about the experimental drug's safety profile. Here's the passage from Batta's email, sent to Parent Project Muscular Dystrophy, that stood out to me: The story that appeared on the investor website highlighted the point that a small number of boys in the clinical trial programme were hospitalised due to thrombocytopenia (a decrease in the number of platelets or small cells that help blood to clot) and proteinuria (excessive protein in the urine). The boys have all recovered, following withdrawal of drisapersen and appropriate medical management. [My emphasis in bold.] See if (GSK) is in our portfolio Toxicity requiring treatment with drisapersen to be stopped is not good thing! Drug withdrawal is more serious than if treatment was merely interrupted. Here is the original post: More Safety Questions for Glaxo DMD Drug; Sarepta Meets With FDA Soon … Continue reading →

Eau Claire (WQOW) - Saturday was a day for moving to the music in Menomonie. A Zumba dance benefit was held for 11 year-old Brett Boettcher, a fifth grader with a degenerative muscular condition. We went to "Shake it for Duchenne" to watch people dance like crazy for a cure. Brett Boettcher's body moves to the beat of a slightly different drum than most kids his age. "It's a lethal, genetic disease that there is no cure, there is no real treatment for right now," said Bart Boettcher, Brett's father and Menomonie High School's Athletic Director. The Menomonie fifth grader has Duchenne Muscular Dystrophy, a genetic disease that causes his muscles to deteriorate. "Brett was diagnosed when he was five years old. We noticed actually at little league baseball that something was definitely wrong. He wasn't able to get to first base real quickly," Bart explained. Saturday, at Menomonie Middle School, a Zumba dance benefit helped raise money for Parent Project Muscular Dystrophy. The non-profit group works to find a cure for the disease. "He's able to walk about a block. So after a block, we either have to put him in a wheelchair, jump on a back, something like … Continue reading →

HACKENSACK, N.J., March 1, 2013 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD) will provide $100,000 to Dr. Stanley Nelson of the University of California, Los Angeles, to examine the genomes of 40 boys with Duchenne muscular dystrophy (Duchenne) who are either very mildly affected or very severely affected. The goal is to identify changes in genes other than dystrophin that may affect the course of the disease. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) "We know that even boys who have exactly the same mutation in the dystrophin gene can have different disease courses," said Dr. Nelson. "It is very likely that other mutations or DNA variants in different genes may play a role in addition to dystrophin." Dr. Nelson will use a relatively new technique known as "exome sequencing" to look only at the parts of the genetic code that spell out the recipe for proteins, known individually as "exons." Because the exons of the genome make up less than 1% of the total genetic material, this method provides a far more practical way to look at the coding regions (genes) for the about 20,000 human proteins without having to sequence the participants' entire genome. For this project, Dr. Nelson will obtain blood samples primarily … Continue reading →

With an incidence of 1 out of 3,500 male births, Duchenne muscular dystrophy is one of the most common of the orphan or rare diseases. But, the effects of the disease, which causes death by the average age of 25, are some of the most destructive according to experts. Orphan or rare diseases are defined in the United States as an illness that affects fewer than 200,000 people throughout the country. Duchenne is a subset of muscular dystrophy that occurs when a person has a genetic defect in which they cannot produce a protein that is present in muscle tissue, called dystrophin. It has the same worldwide incidence as it does in the United States, and may even have the same proportion of cases in the animal kingdom as well. 16 Photos Duchenne's patients are diagnosed around 3 or 4 years old, and normally don't show symptoms before they start exhibiting muscle weakness and are unable to keep up with their peers. "It's pretty devastating," Eric Hoffman, the director of the Center for Genetic Medicine Research at Children's National Medical Center, said to CBSNews.com. "These boys are born normally, and don't show weakness until early school age. We just do … Continue reading →