Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).
Cardiac disease, particularly dilated cardiomyopathy and heart failure, is the major cause of mortality in patients with muscular dystrophy and is present in most boys with Duchenne muscular dystrophy and approximately 70 percent of those with Becker muscular dystrophy. These are the two common forms of muscular dystrophy caused by defects in a gene called dystrophin.
Adult stem cells taken from bone marrow are the "shooting stars" of their field. Many research scientists have been speculating that the cells might be able to pass through the blood into diseased organs and replace defective tissue. Such cells are seen as the potential key to the treatment of certain muscle diseases.
A common chemical used in the manufacturing and pharmaceutical industries can repair damage to cardiac muscle cell membranes and prevent heart failure in mice with the genetic mutation that causes Duchenne muscular dystrophy, according to scientists at the University of Michigan Medical School.
Skeletal muscles naturally repair themselves very efficiently after injury. But when they don’t, otherwise successful recovery following damage from overuse during exercise, surgery or trauma can be stymied. Furthermore, as we age, muscle repair slows noticeably, and in Duchenne Muscular Dystrophy and other degenerative muscle diseases, normal repair functions can’t cope with disease progression.
A new study from Karolinska Institutet in Stockholm shows that hundreds of genes in the thigh muscle are activated in regular cycle training. The study also reveals that great differences in training response may be due to the ability in some people to activate their genes much more forcefully. The study is published May 2 in FASEB Journal.
Corticosteroids can be beneficial in the treatment of Duchenne muscular dystrophy and can be offered as a treatment option, according to the American Academy of Neurology and the Child Neurology Society in a new practice guideline published in the January 11 issue of Neurology, the scientific journal of the American Academy of Neurology.
Researchers at the University of Pennsylvania School of Medicine report a novel strategy for stimulating the production of utrophin – an important muscle protein in young mice – for muscular dystrophy therapy.
MDA-supported researchers at the University of Washington-Seattle have delivered the gene for the dystrophin protein to all voluntary muscles with a single intravenous injection in mice with Duchenne muscular dystrophy (DMD).
The Muscular Dystrophy Association has announced it has awarded $1.6 million to Asklepios, a biotechnology company closely allied with the University of North Carolina at Chapel Hill, to develop gene therapy strategies for Duchenne muscular dystrophy (DMD), a fatal, childhood-onset disease.
A research team funded by MDA has discovered a naturally occurring genetic change (mutation) in humans that dramatically increases muscle size and strength.
Some 35 neuromuscular disease experts, along with biotechnology and government representatives, will meet Friday and Saturday at the Westin La Paloma here to develop a large-scale network to test potential treatments in muscular dystrophy
Despite a roller-coaster ride of ups and downs during the past 15 years, gene therapy has continued to attract many of the world's brightest scientists. They are tantalized by the enormous potential that replacing missing genes or disabling defective ones offers for curing diseases of many kinds.
Grip strength in the dominant hand and fat-free body mass (made mostly of muscle) increased when children with Duchenne muscular dystrophy (DMD) took the dietary supplement creatine at a dose of 0.1 grams per kilogram of body weight per day for four months.
The muscle supplement creatine can be safely taken by children with neuromuscular diseases, but does not improve strength or muscle mass, according to research that will be presented at the American Academy of Neurology 56th Annual Meeting in San Francisco, Calif., April 24 – May 1, 2004.
A team including MDA grantee Gordon Lynch in the Department of Physiology at the University of Melbourne in Victoria, Australia, found that a biological signaling pathway may help explain why mice with Duchenne muscular dystrophy (DMD) can regenerate their muscles much better than can humans with the same disease.
A consortium which includes Oxford researchers has won £1.6m in government funding for ground-breaking research into a possible cure for muscular dystrophy.