Duchenne Muscular Dystrophy (DMD)

Utrophin Gets In

MDA grantee James Ervasti and colleagues at the University of Minnesota-Twin Cities in Minneapolis have found that a protein known as utrophin, injected into mice lacking the dystrophin protein and showing a disease resembling Duchenne muscular dystrophy (DMD), conferred significant benefits.

The experiments Ervasti and colleagues describe online May 26, 2009, in PLoS Medicine, are the first to show benefit from the direct injection into DMD mice of utrophin protein, rather than utrophin genes or gene modifiers.

Fighting the Fire

A protein called osteopontin has been implicated as a cause of some of the detrimental inflammation and scarring ("fibrosis") of muscle tissue that takes place in Duchenne muscular dystrophy (DMD). Eliminating osteopontin was beneficial to mice with a DMD-like disease, and the researchers concluded that reducing osteopontin should be investigated as a possible therapy for DMD.

DMD, MG Research: Spotlight on Prednisone

Some interesting findings about prednisone’s effect on behavior in DMD, and on drugs that may alter its usage in myasthenia gravis, were part of the 61st annual meeting of the American Academy of Neurology (AAN), held recently in Seattle.

DMD STUDY

Daily prednisone led to better behavior than weekly, high-dose prednisone

Biology Prize

On May 3, 2009, molecular biologist Louis Kunkel at Children's Hospital in Boston and Harvard University, and biophysicist Kevin Campbell at the University of Iowa, received the prestigious March of Dimes Prize in Developmental Biology. The prize includes a $250,000 cash award.

DMD: Restarting Muscle Development?

A protein called laminin 111 had a marked therapeutic effect in mice that lack the dystrophin protein and have a muscle disease resembling human Duchenne muscular dystrophy (DMD), say researchers at the University of Nevada School of Medicine.

Patching the Membrane

Scientists in the United States and Japan say they've identified a previously unknown but crucial step in a natural muscle-cell repair process that could have implications for the treatment of muscular dystrophies, particularly those in which membrane defects are implicated.

Plugging a Leak

Investigators conducting experiments in mice with a disease resembling Duchenne muscular dystrophy (DMD) believe they’ve uncovered a new strategy to protect against muscle damage and improve strength in this disease.

Andrew Marks at Columbia University in New York coordinated the team, which included researchers from Montpellier (France) University and other institutions in Montpellier. They published their findings in the March issue of Nature Medicine.

DMD Research: Doubts About EKGs

Kid getting EKG
An electrocardiogram (EKG) transmits information about heart rhythms, in the form of electrical signals, to a computer. Researchers warn that the test “should not serve as a basis for decisions regarding treatment” in people with DMD.

MDA's Voice for Change

Palliative care for kids, transition services for young adults, and genetic discrimination are just some of the projects being worked on by MDA’s Advocacy program, under the energetic direction of MDA Vice President Annie Kennedy.

Following are some highlights of these projects.

Palliative care in pediatrics

DMD: Gene-Changing 'Cocktail'

Scientists at Children's National Medical Center in Washington, Carolinas Medical Center in Charlotte, N.C., and the National Center of Neurology and Psychiatry in Tokyo, have successfully treated dogs with a disease closely resembling Duchenne muscular dystrophy (DMD) , using a molecular treatment strategy called "exon skipping." The strategy is simultaneously under development in human patients.

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