Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.

MDA opens center for Lou Gehrig's disease

The University of Rochester Medical Center in New York state has been designated as the site of a new MDA/ALS center. The center is the 32nd facility to receive the designation from the Muscular Dystrophy Association’s ALS Division.

13 Aug 2004

Proof of principle that it is possible to deliver new genes body-wide to all the muscles of an adult animal

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).

29 Jul 2004

$1.6 million to develop gene therapy strategies for Duchenne muscular dystrophy, a fatal, childhood-onset disease

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.

23 Jul 2004

Naturally occurring genetic change in humans that dramatically increases muscle size and strength

A research team funded by MDA has discovered a naturally occurring genetic change (mutation) in humans that dramatically increases muscle size and strength.

28 Jun 2004

Mighty mouse gene works the same way in humans

By studying the genes of a German child born with unusually well developed muscles, an international research team has discovered the first evidence that the gene whose loss makes "mighty mice" also controls muscle growth in people.

26 Jun 2004

Muscular German boy sheds new light on muscle development and genetics

By studying the genetic make up of a German boy born with unusually well developed muscles, an international research team has discovered the first evidence that the gene whose loss makes "mighty mice" also controls muscle growth in people.

24 Jun 2004

Large-scale network to test potential treatments in muscular dystrophy

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

10 Jun 2004

Research may help treat muscular dystrophies and other muscle diseases caused by glycosylation defects

Expressing high levels of a sugar-adding protein known as LARGE in mice that lack the protein can prevent muscular dystrophy in these animals, according to studies by researchers at the University of Iowa Roy J. and Lucille A. Carver College of Medicine.

7 Jun 2004

Breakthrough in gene therapy

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.

3 Jun 2004

MDA researchers find creatine helps in Duchenne dystrophy

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.

25 May 2004

Closer to understanding the function of a protein linked to an inherited form of the movement disorder dystonia

The protein, torsinA, is defective in patients with DYT1 dystonia, an inherited condition that causes uncontrollable movements in the limbs and torso. Learning what torsinA does could be an important step toward developing a treatment for the disorder.

19 May 2004

Lou Gehrig’s disease gene discovered

A gene for a rare juvenile-onset form of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) has been isolated in families in the United States, Belgium and Austria, the Muscular Dystrophy Association (MDA) announced today.

29 Apr 2004

The muscle supplement creatine is safe, but not effective, in pediatric neuromuscular disease

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.

27 Apr 2004

Researchers have discovered a genetic mutation associated with an inherited form of motor neuron disease

Researchers have discovered a genetic mutation associated with an inherited form of motor neuron disease in which symptoms first appear in childhood or young adulthood. The finding is slated for publication in the American Journal of Human Genetics.

20 Apr 2004

Researchers pinpoint pathway to muscle regeneration

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.

12 Apr 2004

University of Florida researchers have used a common gel to successfully deliver gene therapy to the diaphragm

The technique, described in the current issue of Molecular Therapy, could eventually lead to a method to correct genetic conditions in humans that cause diaphragm weakness and respiratory failure -- a leading cause of death in tens of thousands of patients with forms of muscular dystrophy, including Pompe’s disease. Thousands of Americans with muscle-weakening diseases are placed on ventilators each year, according to the Muscular Dystrophy Association.

7 Apr 2004

Oxford Biomedica to win Department of Health gene funding

Oxford spinout Oxford Biomedica was yesterday awarded £0.5 million by the Department of Health to conduct the first British gene therapy haemophilia trial.

31 Mar 2004

£1.6m for research into a possible cure for muscular dystrophy

A consortium which includes Oxford researchers has won £1.6m in government funding for ground-breaking research into a possible cure for muscular dystrophy.

30 Mar 2004