2006 could prove to be a breakthrough year in the treatment of Muscular Dystrophy.
Two human trials are now underway, both focusing on the gene with the defective protein that causes muscle to waste away. At the University of Utah, 5 year old Jack is getting a drug scientists hope will restore muscle function by bypassing the genetic defect. And at Columbus Children's Hospital, 8 year old Andrew is getting experimental therapy designed to replace parts of the abnormal gene. Researchers are hoping they'll see a glimpse of that progress in the boys.
"I'm the ultimate optimist I admit, I think we can do something about this disease that's why we're here!" said gene therapy researcher Jerry Mendell.
Researchers in both studies say they should have early results in the next few months. The studies focus on a specific type of MD called Duchenne which is the most severe and affects one out of every 3,500 hundred births.
Study locations include: Children's Hospital Medical Center in Cincinnati, Ohio, Children's Hospital of Philadelphia in Philadelphia, Pennsylvania, the University of Utah, in Salt Lake City, Utah, and Columbus Children's Hospital in Columbus, Ohio.