MDA collaborates on clinical trial of a gene-skipping drug for Duchenne Muscular Dystrophy

TAMPA - There is exciting news about a potential therapy for a devastating neuromuscular disease. A clinical trial now underway is showing promise in treating Duchenne Muscular Dystrophy.

In the early months, happy parents may see a normal baby, but by age three, problems may start. In video from YouTube you can see the the progression of the neuromuscular disease.

"Duchenne Muscular Dystrophy is a disease of muscle. It's hereditary and because of that it's found in boys only."  Neurologist Allan Weiss runs the MDA clinic at St. Anthony's Hospital in St. Petersburg. He says most boys with this neuromuscular disease won't live beyond their twenties. "There really aren't any good treatments. Sometimes we use prednisone but that is not an answer from a long term stand point."

But this young boy is one of 12 young men with Duchennes participating in a landmark trial of an Exon skipping drug - that basically takes the area of a gene that is blocked or defective and skips over it.

The drug that does the skipping is given intravenously every week for 24 weeks. The trial includes muscle biopsies, tests and the weekly infusions

Dr. Weiss says, "We're hoping this will make the disease progress much slower and it may not be as severe. As a result, the prognosis would be much better. Obviously a person's life span would be much longer than it is now."

The Muscular Dystrophy Association is helping to fund the trial which is why we need your help this Labor Day weekend. 

Please join us for the annual MDA telethon Sunday. The telethon starts at 6pm and runs until midnight right here on your ABC Action News station.

Print this article Back to Top

Comments