Dublin boy paves the way for new muscle disease treatment – 13WMAZ.com

Karli Barnett, WMAZ 5:06 AM. EST February 27, 2017

A Dublin boy has become a pioneer in how doctor's treat a common form of Muscular Dystrophy.

We first shared Addison Jones story back in 2015 when he started taking part in a medical study. Now, hes helping to make the treatment possible for other children like him.

When we met Jones two years ago, he loved to race, and not much has changed.

Addison in his own race of sorts -- one against a rapidly progressing disease.

He has Duchenne Muscular Dystrophy, the most common kind. It typically affects boys and as he gets older, it's going to get harder to walk and stand.

But two years ago, his grandfather John and his family found hope. Doctors selected him as one of 10 boys for a trial drug called Eteplirsen.

Every Friday, Addison and his family make the trip all the way from Dublin to Children's Healthcare of Atlanta for his treatment. What started out as a trial, has now been FDA approved.

Research shows Eteplirsen slows the disease's progression allowing children to walk for longer.

Children with Duchenne's are usually wheelchair dependent by their early teens. Addison can still walk, but uses a scooter for long distances.

He is getting up out of chairs [that] he couldn't before. He went with me the other day to Home Depot and said I want to walk, said his grandfather John Dykes.

That might not sound significant, but its a huge victory for them.

We now have the first ever approved drug by the FDA, so this is a very exciting and promising time for our patients, said Dr. Han Phan.

Phan is a pediatric neurologist and says even though there is no cure for DMD, it is progress.

So we hope that with the approval of this drug, there will be more to come in the future, said Phan.

That's what we keep telling him. He's one of the pioneers with this drug, said Dykes, to the point that he was in Washington [D.C.] with the FDA and was actually part of them approving it and part of the reason they approved it.

It feels good to help other people like me, said Jones.

The weekly trip to Atlanta and the portal in his chest to deliver the drug took some getting used to, but now the fourth grader looks forward to his visits.

Because I get my medicine and it makes me stronger, said Jones.

It's a good thing because he has the same lofty goal today -- as he did the first time we spoke -- to be the President of the United States.

What may have seemed like an uphill race is now speeding closer to the finish line.

Addison will continue this treatment for the rest of his life.

Eteplirsen will now be available in more hospitals across the country, and in the last two weeks, the FDA has since approved another drug for Duchenne's that treats the symptoms, rather than the underlying cause of the disease.

( 2017 WMAZ)

Continued here:
Dublin boy paves the way for new muscle disease treatment - 13WMAZ.com