Myopathy Press Releases

One of the biggest challenges in developing useful gene therapy is finding a way to get the beneficial gene into enough cells of the body to effectively treat the disease. Now, researchers have shown in rodents that a virus called adeno-associated virus 8 (AAV8) can effectively deliver a gene to all the skeletal muscles of the body. If it works the same way in humans, this virus-based approach may allow the first effective gene therapy for muscular dystrophy (MD) and similar diseases.

Patients with a painful and debilitating muscle disease called dermatomyositis showed dramatic improvement on a treatment regimen of intravenous immune globulin (IVIG) during a recent double-blind, placebo-controlled trial. The study, which was conducted at the National Institute of Neurological Disorders and Stroke (NINDS), will be published in the December 30 issue of the New England Journal of Medicine.
Fact Sheet

Researchers have revealed what may be a totally new cause for muscular dystrophy (MD). A recent study shows that a protein defective in two types of late-onset MD plays a critical role in the normal repair of muscles.
Fact Sheet