Category Archives: Muscular Dystrophy Treatment

The University of Virginia Medical Center is making strides in finding a treatment for myotonic dystrophy, which the most common type of muscular dystrophy. The potential treatment is an experimental drug currently used to treat other conditions, such as rheumatoid arthritis. In lab tests, mice with myotonic dystrophy that were given this drug had better muscle function and improved strength. Doctors are hopeful that this could also be applied to humans. "The goal here would be to block the inflammation that is occurring in the muscles because it appears that that inflammation, the longer it goes on in these chronic diseases, the more it leads to muscle degradation and muscle break down," said Erin Foff, assistant professor at the UVA Medical Center Department of Neurology. The drug will now need to go through clinical trials with myotonic dystrophy patients. CHARLOTTESVILLE, Va., March 17, 2015 A doctor who was one of the discoverers of the gene responsible for myotonic muscular dystrophy has now identified a therapeutic that could slow progression of muscle damage and muscle dysfunction associated with the disease issues that cause patients significant disability and deterioration in quality of life. The potential treatment is an experimental drug currently being … Continue reading →

Duchenne Muscular Dystrophy Symptoms Treatment aims to control Duchenne Muscular Dystrophy Symptoms to improve quality of life. Previously, he used to fall for almost 3 to 4 times, but now we ha... By: Neurogen Brain and Spine Institute … Continue reading →

WASHINGTON, March 12, 2015 /PRNewswire-USNewswire/ --The Muscular Dystrophy Association's chief executive today opened its 2015 Scientific Conference in Washington by declaring to the more than 400 physician-scientist attendees that MDA's number one mission priority is to accelerate discovery of treatments and cures for the families it serves. "This conference underscores two foundational commitments our Board recently affirmed in our new Mission 2020 plan," said MDA President and CEO Steven M. Derks. "First, MDA wants to continue to be the research leader in the neuromuscular and motor neuron space and second, MDA believes it needs to be an even more impactful and effective convener, collaborator and knowledge broker than in the past." MDA's next generation of clinical and research changes are part of a bold plan to support families "solutions for them can't come quickly enough," Derks said. His remarks come as MDA takes steps to refocus its basic and translational research program and capitalize on its position to advance frontline discoveries. The CEO disclosed a five-year plan that includes: "We want more answers (for families) faster, pretty simple," Derks said. "It's why we wake up in the morning and it's why we're all here today." MDA is leading the way … Continue reading →

CHICAGO, March 10, 2015 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association will hold its 2015 Scientific Conference at the Washington Marriott Wardman Park in Washington, D.C., March 11-14. The meeting is the preeminent gathering of the nation's leading scientists, researchers and professionals specializing in neuromuscular disease research and care, designed to accelerate discoveries and drug development for families fighting neuromuscular diseases, for which there are currently no cures. The conference is part of MDA's annual conference series, which hosts clinical gatherings and scientific research gatherings in alternate years. "One key strategy to accelerating lifesaving and life-enhancing treatments and cures for neuromuscular and motor neuron diseases is to ensure collaboration among the nation's top thought leaders in our scientific community," said Steven M. Derks, MDA President and Chief Executive Officer. "I'm proud that, in addition to the hundreds of different research projects we fund, MDA, through our Scientific and Clinical Conferences and symposia, continues to be a convener and facilitator for researchers nationwide to share ideas, best practices, and novel strategies.Shared knowledge through scientific discoveries and failures speeds progress for the families we serve. Together, we are accelerating frontline discovery and bringing urgently-needed answers to children and adults affected by muscular dystrophy, … Continue reading →

WTAE is proud to support the Pittsburgh Muscle Team, benefiting the Muscular Dystrophy Association. Emceed by Pittsburghs Action News 4 anchors Wendy Bell and Andrew Stockey, Muscle Team is a high profile social event to benefit the Muscular Dystrophy Association, where business people have the opportunity to mix and mingle with their favorite athletes! The concept of Muscle Team is to gather top athletes and pair them with a MDA Goodwill Ambassador, a local child living with a neuromuscular disease. At Muscle Team, participants get the chance to meet and greet the Muscle Team Coaches, Players, Goodwill Ambassadors and top business executives. The evening consists of fun events, interactive entertainment, a variety of food and beverage stations, and is highlighted by extensive media coverage, live and silent auctions, and unique sports memorabilia. Visit the muscle team website: http://www.pittsburghmuscleteam.org MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education. MDA is one of the world's leading national health agencies, fostering research and patient care. MDA's programs are funded by individual and private contributors. The Association does not seek or … Continue reading →

Stem Cell Sound Bites: Duchenne Muscular Dystrophy Visit: http://www.uctv.tv/) Carrie Miceli and Stanley Nelson of UCLA describe their efforts to use stem cell-based strategies to find a drug treatment for d... By: University of California Television (UCTV) … Continue reading →

A leading scientist from Newcastle University has won a prestigious award for her pioneering work on neuromuscular diseases. Professor Kate Bushby received the 2015 EURORDIS Scientific Award for her research into rare inherited genetic conditions. I am delighted to have received this award: a real sign of recognition that the John Walton Muscular Dystrophy Research Centre is a world leader in the field of rare diseases, said Prof. Bushby, and honorary consultant geneticist at Newcastle Hospitals NHS Foundation Trust, who is based at the Centre for Life. The award is also a real accolade for the work of all our many partners in Europe and worldwide and an indication that collaboration is essential for advancing research in rare diseases. Genetic neuromuscular diseases are disorders that affect nerves which control the bodys voluntary movements, and a number of such conditions are extremely rare. Newcastles research centre is at the forefront of work on the conditions, with its team collaborating with experts and patient organisations from around the globe to help improve diagnosis, care and treatment for patients. Nominations for the EURORDIS Scientific Award came from the public, with members of the rare disease community and beyond then having their say on … Continue reading →

Chicago, IL (PRWEB) February 27, 2015 Muscular Dystrophy Association research grantee Charles Gersbach, Assistant Professor of Biomedical Engineering at Duke University, recently announced a potentially game-changing advance in gene modification for boys and young men with Duchenne muscular dystrophy (DMD). The results were published Feb. 18 in Nature Communications, and Gersbach will discuss their implications at MDA's 2015 Scientific Conference, to be held March 11-14 in Washington, D.C. Background: Gersbach and team are investigating a new genetic therapeutic technique that has the potential to treat more than half of patients fighting DMD and could be developed as a permanent, one-time treatment. Known as CRISPR-Cas9 genome editing, the strategy targets a large area of the dystrophin gene (DNA) and is designed to cause production of shorter-than-normal, but still functional, dystrophin protein in muscle tissue. If successful in humans, it could prolong function and increase longevity. Other promising experimental DMD treatments such as the experimental drugs eteplirsen and drisapersen are currently being tested in clinical trials, but because these other treatments target dystrophin RNA rather than DNA, they would not result in a permanent correction and would require repeated treatment over a patients lifetime. Ever since MDA-supported researchers identified flaws in the … Continue reading →

A LEADING North-East scientist has won a prestigious award for her pioneering work into neuromuscular diseases. Professor Kate Bushby, from Newcastle University, has received the 2015 EURORDIS Scientific Award, which recognises her outstanding research into rare inherited neuromuscular genetics. Nominations for this award were received from the public, giving an opportunity for everybody in the rare disease community and beyond to have their say on who should get the top accolade. Professor Bushby, who is an expert in neuromuscular genetics at Newcastle University, is director of the John Walton Muscular Dystrophy Research Centre, based at Newcastles International Centre for Life. She is also honorary consultant geneticist at Newcastle Hospitals NHS Foundation Trust. The research centre is at the forefront of developments in neuromuscular diseases as its team work with experts and patient organisations from around the globe to help improve diagnosis, care and treatment for patients. Professor Bushby said: I am delighted to have received this award: a real sign of recognition that the John Walton Muscular Dystrophy Research Centre is a world leader in the field of rare diseases. Genetic neuromuscular diseases are disorders that affect nerves that control the bodys voluntary movements, and a number of conditions are … Continue reading →

Tracey Romero, Sports Medicine Editor, Philly.com Posted: Friday, February 27, 2015, 2:50 AM The word rare connotes something so unique and scarce that it becomes more valuable. When it comes to rare diseases though, the small patient populations usually means the disease has to fight harder for attention and funding. On World Rare Disease Day, which will be held on Saturday, February 28 this year, patients and their families as well as scientists, physicians and researchers will try to draw more awareness to rare diseases in an effort to improve access to treatment and to drive more research to be done. Although rarer than say heart disease or breast cancer, these diseases still have a deep impact on people both in our country and worldwide. According to the Center for Orphan Disease Research and Therapy at the University of Pennsylvania, orphan/rare diseases represent a collection of disorders that affect fewer than 200,000 individuals for any single disease type, yet there are more than 7,000 distinct orphan diseases. In all, over 25 million people in the United States are afflicted by orphan diseases. Most rare diseases are genetic and many of them present in early childhood. About 30 percent of children … Continue reading →