Drug showing promise with muscular dystrophy

By Misti Crane

The Columbus Dispatch Friday October 5, 2012 9:01 AM

Parents of boys with Duchenne muscular dystrophy now have even more reason for hope.

The company that makes an experimental drug designed to treat about 13percent of the approximately 12,000 U.S. boys with the disease has released new results from a small but important study that shows significant benefit from the drug eteplirsen.

Dr. Jerry Mendell, director of the Center for Gene Therapy at Nationwide Childrens Hospitals research institute, is the lead researcher on the study of 12 boys with the debilitating, and ultimately deadly, disease.

He and the company, Sarepta Therapeutics, previously shared optimistic news after observing the boys health about nine months into treatment with a weekly infusion of the drug.

Now, they say they have seen significant benefit after almost a year. Sarepta Therapeutics said this week that the drug increases dystrophin, a vital protein missing in boys with the genetic disorder. It also slowed progression of the disease, as measured by how far the boys could walk in six minutes.

The four boys who had the strongest dose for 48 weeks were able to walk an average of about 23 yards farther than when they began taking the drug. Those who received a placebo for 24 weeks followed by 24 weeks of treatment lost about 74 yards.

The 97-yard difference is profound in a disease that forces most boys into wheelchairs before they reach their teens.

What remains unclear is how the drug performs in the long term and whether there are side effects.

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Drug showing promise with muscular dystrophy