One Charlotte boy hopes to help find a cure for rare muscle dise - KFVS12 News & Weather Cape Girardeau, Carbondale, Poplar Bluff

One Charlotte boy hopes to help find a cure for rare muscle disease

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CHARLOTTE, NC (WBTV) -

Brady Williams has an infectious personality.

The Charlotte boy has a lot to be happy about.

"I'm faster and I can open drinks," said Brady.

That same enthusiasm can be seen in his mother. Martha Williams is convinced that the 12-year-old is being helped by the experimental drug which counteracts his Duchene muscular dystrophy.

"I've seen a big difference," said Williams.

"When we first started he would always have to have his wheelchair from the car to get up here and a lot of times he walks now.'

Brady is being treated with an experimental drug called Eteplirsen for arare muscle deteriorating disease known as Duchenne muscular dystrophy, which is visibly helping him with his condition by making him stronger.

His ability to walk has not deteriorated in the two years he has been on the drug, whereas many boys with the disease would be in wheelchairs by his age

"There really is no cure for the disorder and so that's why we are doing the research to find new therapies," said Dr. Susan Sparks with Levine Children's Hospital.

Brady is one of 12 boys in the entire country taking part in a clinical study.

Dr. Sparks said the drug works by helping the body to produce a protein called dystrophin that is lacked by people who have Duchenne muscular dystrophy.

"He has been getting this medication now for 80 plus weeks and I've noticed that he is still ambulatory," said Dr. Sparks. 

"And for a 12 year old boy with Duschenne that's unusual to be still be able to walk without needing a wheelchair."

Given Brady's remarkable success with the clinical trial, the family is hopeful the drug will soon get FDA approval.

"I'm hoping that everything we've been through and the other kids and their families have been through is going to make it worthwhile for everybody else to benefit from it," added Williams.

The drug Eteplirsen just received a two year extension of the trial.  Eteplirsen is not a cure and would be a lifelong treatment.

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