30-person trial will test the safety, tolerability and pharmacokinetics of the experimental drug HT-100 in boys with Duchenne muscular dystrophy

posted on July 30, 2013 - 1:51pm
Update (Jan. 6, 2014): In a Dec. 23, 2013, announcement, Halo said the U.S. Food and Drug Administration (FDA) had placed this trial on "clinical hold" because of some adverse events that occurred in dogs being treated with HT-100. No further dosing of patients will occur until this issue has been resolved.

Three recent MDA-supported studies investigate the nature of cardiac problems in Duchenne/Becker MD and propose possible treatment avenues

posted on June 6, 2011 - 4:30pm
In both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), deterioration of the heart muscle, a condition known as cardiomyopathy, is a major cause of disability and death.

A change in the LTBP4 protein gene reduces muscle-damaging signals in mice with a disease resembling LGMD2C

posted on November 9, 2009 - 9:46am
New research has shown that a change in a gene not previously connected to type 2C limb-girdle muscular dystrophy (LGMD2C) modifies the severity of the disease in mice and is likely to do the same in people with this and perhaps with related types of muscular dystrophy.

Can an immune response be rerouted to treat disease?

posted on July 1, 2008 - 1:00pm
QUEST Vol. 15, No. 4
What do inflammatory muscle diseases (myositis), myasthenia gravis, Lambert-Eaton syndrome, and dozens of other disorders like lupus and rheumatoid arthritis, have in common?
posted on July 1, 2007 - 10:32am
QUEST Vol. 14, No. 4
The underlying cause of Duchenne muscular dystrophy (DMD) can be any of a large number of mutations in the gene for the dystrophin protein. One strategy now being tested for overcoming this problem is gene therapy, the insertion of new dystrophin genes. Another is the transplantation of cells that give rise to muscle.
posted on June 1, 2001 - 3:31pm
Usually, inflammation of tissue occurs with an injury or an infection, but it's also an important part of certain neuromuscular diseases.