Muscular Dystrophies

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.

Silencing Toxic Genes

A new gene therapy approach to "silencing" disease-causing genetic information has been developed by researchers at Rutgers University in Piscataway, N.J., and Integrated DNA Technologies in Coralville, Ia.

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.

Possible New Therapy for OPMD?

Scientists in France and the Netherlands recently announced they've identified a promising new strategy that could potentially become a therapy for oculopharyngeal muscular dystrophy (OPMD), a form of MD that primarily weakens the eyelid and throat muscles and also can affect muscles in the limbs.

The strategy involves using an antibody (immune-system protein) derived from llamas. The antibody sticks to abnormally formed protein molecules in muscle cells and keeps them from forming large, damaging clumps.

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.

Building a Better Gene

Displacement of a protein called neuronal nitric oxide synthase (nNOS) from the membrane that surrounds each skeletal muscle fiber appears to be a much more important contributor to exercise intolerance and even cardiac degeneration in some forms of muscular dystrophy than previously recognized.

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