Category Archives: Muscular Dystrophy Treatment

How is Muscular Dystrophy Diagnosed? Muscular dystrophy (MD) is diagnosed through a physical exam, a family medical history, and tests. These might include: For Duchenne and Becker muscular dystrophies, muscle biopsy may show whether dystrophin, a muscle protein, is missing or abnormal, and DNA testing is used to analyze the condition of the related gene. Genetic testing also is available for some forms of muscular dystrophy. There is no cure for any form of muscular dystrophy, but medications and therapy can slow the course of the disease. Human trials of gene therapy with the dystrophin gene are on the near horizon. For instance, scientists are researching ways to insert a working dystrophin gene into the muscles of boys with Duchenne and Becker muscular dystrophies. Researchers are investigating the potential of certain muscle-building medicines to slow down or reverse the progression of muscular dystrophy. Other trials are looking into the effects of the dietary supplements creatine and glutamine on muscle energy production and storage. Symptoms often can be relieved through exercise, physical therapy, rehabilitative devices, respiratory care, and surgery: In some cases, disease progression can be slowed or symptoms relieved with medication: Medications also can be prescribed for some muscular dystrophy-related … Continue reading →

1 November 2013 Last updated at 09:20 ET By Marianne Taylor BBC Scotland news Families of children with a devastating muscle-wasting condition have welcomed funding for a new medical expert. Duchenne muscular dystrophy, which affects about 200 children and young people in Scotland, is incurable and causes severe disability. A shortage of experts makes it difficult for potential new treatments to reach those with the condition. Parents said the funding would help their children lead more fulfilling lives. We need to ensure any treatments developed by scientists make it to the people who desperately need them Duchenne, which mostly affects boys, causes muscles to weaken and waste over time, leading to increasing and severe disability. Few born with the condition - which affects about one in 3,600 boys - currently live to the age of 30 due to damage to heart and breathing muscles. A new funding partnership between the Chief Scientist Office and charities Muscular Dystrophy Campaign and Action Duchenne, will provide 75,000 a year for three years for a medical training fellow to research potential treatments and work directly with children. It is hoped the new expert will be in post by autumn 2014. Originally posted here: Funding … Continue reading →

PUBLIC RELEASE DATE: 31-Oct-2013 Contact: Malene Bech Vester-Christensen mbxc@novonordisk.com 45-22-15-25-89 University of Copenhagen About 3,000 people in Denmark suffer from one of the serious muscle-related diseases that come under the heading of muscular dystrophy. Some patients diagnosed with muscular dystrophy die shortly after birth, others become severely retarded and develop eye problems, while certain groups are confined to life in a wheelchair. Common to all muscular dystrophy sufferers is the difficulty of their muscle cells to attach themselves to each other and to the surrounding tissue. However, little is actually known about the root causes of the disease. New basic research from University of Copenhagen now offers insight into previously unknown facts about muscular dystrophy that may improve future diagnosis and treatment of the disease. The findings have just been published in the scientific journal PNAS (Proceedings of the National Academy of Science). "Our new research findings may shed light on some of the cellular processes that take place in connection with, for example, muscular dystrophy. This is important information because it is crucial for us to gain as detailed an understanding as possible about the individual cell components. Although the journey from the current basic research to any potential … Continue reading →

Newswise Scientists in The Research Institute at Nationwide Childrens Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease. Gene therapy is among the most promising treatment options for such genetic disorders as muscular dystrophy, congenital blindness and hemophilia. Scientists also are investigating gene therapy as a cure for some cancers, neurodegenerative diseases, viral infections and other acquired illnesses. To get the therapeutic gene into cells, researchers have turned to viruses, which deliver their genetic material into cells as part of their normal replication process. Time and time again, these efforts have been thwarted by the bodys own immune system, which attacks the viral vector. The therapeutic genes arent delivered and disease rages on. Now, a team led by Louis G. Chicoine, MD, Louise Rodino-Klapac, PhD, and Jerry R. Mendell, MD, principal … Continue reading →

Hereditary condition presents challenges for patients, families PORTSMOUTH There are a lot of muscular disorders that can occur in life, but doctors say the condition known as muscular dystrophy is hereditary and will begin to present in childhood. Muscular dystrophy is marked by a progressive weakening and wasting of muscles. It can affect the heart and other organs, and breathing and swallowing. Limbs draw inward and become fixed (contracture). Eventually, the patient will require a wheelchair. There is no cure and the disease will ultimately lead to death. Treatment is palliative, for comfort and to try to slow the disease progression. Dr. Douglas Black, a neurologist at Portsmouth Regional Hospital, said that, as an inherited condition, muscular dystrophy can present in infants. Called the floppy baby syndrome, infants carrying the trait will have very low muscle tone. "When we think about the disease in children it is the Duchenne version, as in the condition the Jerry Lewis telethon is about," Black said. "It is the most common and children afflicted rarely live past age 20. There is Becker muscular dystrophy, a milder version. Becker may not present until the person is a young adult and the life span is generally … Continue reading →

PHOENIX - The Muscular Dystrophy Association will hold its Taste of the Town fundraising event at Scottsdale Quarter on Friday. Robert Reed was diagnosed with muscular dystrophy at age 8. He received care at the Barrow Neurological Institute at Phoenix Children's Hospital. "When I was diagnosed, my family didn't have medical insurance, so MDA was there to provide free doctor care and helping me get diagnosed and getting the treatment that I needed," he said. Thanks to MDA, Robert also attended summer camp and discovered wheelchair basketball. He's a member of the Banner wheel chair Suns and also coaches the junior team. Now 22, Robert is an advocate for other kids and families battling the disease. "As a kid, I didn't necessarily understand what was going on, but for my parents, it was really important to be able to talk to other parents that were going through the exact same thing," he said. Taste of the Town is one of the biggest fundraising events MDA hosts locally. It will feature over 25 food and wine vendors, live music and a silent auction. "What they're doing in this event and raising money.. it will go to help not only myself, but … Continue reading →

HACKENSACK, NJ and CAMBRIDGE, MA--(Marketwired - Oct 17, 2013) - Parent Project Muscular Dystrophy (PPMD) and Sarepta Therapeutics, Inc. (NASDAQ: SRPT) today announced a new nationwide program from PPMD to assist individuals with Duchenne muscular dystrophy in accessing genetic testing. The program will be administered by PPMD, a leading voice for patients and families impacted by Duchenne. Support for the initiative will be provided by Sarepta, a developer of innovative RNA-based therapeutics. Through the new program, called Decode Duchenne, PPMD will offer genetic testing at no cost to eligible patients who are unable to access testing due to barriers such as a lack of or insufficient insurance coverage. Decode Duchenne is expected to launch in the fourth quarter of 2013 through PPMD's DuchenneConnect, an online resource and registry for patients and families affected by Duchenne and their healthcare providers. For patients with Duchenne, genetic testing is used to identify the genetic mutation and confirm the diagnosis. In addition, genetic testing can be used to determine a patient's eligibility for certain clinical trials. Despite the potential benefits of testing, in some cases patients are unable to access genetic testing through their insurance provider. "We believe all patients with Duchenne should have … Continue reading →

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Parent Project Muscular Dystrophy logo. (PRNewsFoto/Parent Project Muscular Dystrophy) WASHINGTON, Oct. 9, 2013 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD) the leading patient advocacy organization fighting to end Duchenne muscular dystrophy (Duchenne) is proposing a collaboration with the Food & Drug Administration (FDA) to initiate a rare disease benefit/risk pilot program using Duchenne as the initial therapeutic area. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) In making this call for a Duchenne benefit/risk pilot program, PPMD issued a paper, entitled Benefit-Risk Assessments in Rare Disorders: The Case for Therapeutic Development in Duchenne Muscular Dystrophy as the Prototype for New Approaches, that thoroughly explores the benefit/risk paradigms used in both the U.S. and Europe, including patient survey data from a recent PPMD study, and that offers recommendations for ways to modify these frameworks to accommodate the unique needs of rare disease communities. "A robust and functioning system to evaluate and consider the benefits and risks of a potential therapy is critical to the process of reviewing and approving novel therapies," Parent Project Muscular Dystrophy Founding President & CEO Pat Furlong said. "In making such decisions, particularly on therapies intended to treat rare diseases, it is absolutely essential that reviewers and regulators fully understand the perspective of the … Continue reading →

Salt Lake City (PRWEB) October 07, 2013 Fire fighters will be out on the streets this week throughout the Salt Lake Valley asking motorists to help Fill the Boot for the Muscular Dystrophy Association. Beginning today through Saturday, Oct. 12.,every dollar that passing motorists can pitch into the fire fighters boots helps Unified Fire Authority Local1696 fire fighters provide medical treatment and support services to individuals and their families living with neuromuscular diseases in the state of Utah. The International Association of Fire Fighters has been a national MDA partner for more than 50 years and remains committed to the fight to end neuromuscular diseases. Fire fighters are American heroes who make such a difference for the people and families we serve, said MDA Executive Director Jamie Ingersoll. The funds they raise are used for health care services and equipment support, and to help send kids to free MDA summer camps. We are grateful for everything they do. Fill the Boot funds also are used to support some of the 250 worldwide research projects seeking better treatments and cures for the more than 40 neuromuscular diseases covered by MDA, including Duchenne muscular dystrophy, spinal muscular atrophy and ALS. IAFF became … Continue reading →