A team of researchers has recently shown that the administration of sildenafil protects the heart in mice with Duchenne muscular dystrophy.

An investigational antiviral drug currently undergoing human trials in Europe for treating Hepatitis C infections may have potential to reduce muscle cell damage in Duchenne and other forms of muscular dystrophy (MD).

Injecting a gene responsible for making a specific protein into a mouse that’s used as a model for muscular dystrophy can lead to long-term improvements in the animal’s muscle size and strength, a new study shows.

Investigators in The Research Institute at Nationwide Children’s Hospital have identified the role of a protein that could potentially lead to new clinical treatments to combat musculoskeletal diseases, including Duchenne muscular dystrophy (DMD).

Using embryonic stem cells from mice, UT Southwestern Medical Center researchers have prompted the growth of healthy – and more importantly, functioning – muscle cells in mice afflicted with a human model of Duchenne muscular dystrophy.

Using a new type of drug that targets a specific genetic defect, researchers at the University of Pennsylvania School of Medicine, along with colleagues at PTC Therapeutics Inc. and the University of Massachusetts Medical School, have for the first time demonstrated restoration of muscle function in a mouse model of Duchenne's muscular dystrophy (DMD).

In the April 1st issue of Genes & Development, Dr. Bruce Spiegelman (Dana Farber Cancer Institute) and colleagues identify a key genetic component of and possible therapeutic target for Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is characterized by progressive muscle wasting and usually results in death by the age of 30. Although mutations in the dystrophin gene cause DMD, they trigger persistent inflammation, which is probably what intensifies disease progression.