making muscle wasting history

It’s been 20 years since the discovery of the gene that causes the most common type of Muscular Dystrophy – Duchenne MD and patients are still waiting for a cure.

“The mutation causes muscle fibers to pull away from each other and with progressive use of your muscles in these patients it eventually leads to muscle damage, severe muscle damage,” explains Dean Burkin, assistant professor of pharmacology at the University of Nevada, Reno School of Medicine.

Gene therapy to replace the faulty dystrophin gene might be a solution, but Burkin and his colleagues are excited about a simpler and potentially safer approach based on work they are publishing in this week’s Proceedings of the National Academy of Sciences.

“This could be an IV drug for the patients if the work in the mouse models that we've been using translates to human studies,” Burkin says. "That would allow a fair ease of treatment for the patients. We’ve obviously got to do some safety tests and there?s still a few studies that we need to do. But in the field there are a number of drugs including ours that are being developed.

“These patients, especially, have been waiting a long time for new therapies to come about and I think we're at the cusp now.”