Duchenne Muscular Dystrophy (DMD)

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.

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.

DMD Research: Exon Skipping

On Jan. 21, AVI BioPharma of Portland, Ore., announced its experimental compound AVI4658 for the treatment of Duchenne muscular dystrophy (DMD) yielded promising results in a phase 1 clinical trial in the United Kingdom.

DMD Research: Exon Skipping Goes Systemic

AVI BioPharma of Portland, Ore., has started the systemic (through the blood) delivery phase of its clinical trial of AVI4658 in Duchenne muscular dystrophy (DMD). The trial is being conducted in the United Kingdom.

UK Trial: 'Robust Response' to Exon-Skipping Compound Seen

On Jan. 21, AVI BioPharma of Portland, Ore., announced its experimental compound AVI4658 for the treatment of Duchenne muscular dystrophy (DMD) yielded promising results in a phase 1 clinical trial in the United Kingdom.

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