Family Awaits Decision from FDA about Life-altering Drug for Son

A Walker County family is trying to bring national attention to a disease that commonly affects young boys.

Duchenne is a one of the 42 types of Muscular Dystrophy that are out there.

The disease weakens the muscles and can lead to premature death.

The Crawford family is one of many families across the country waiting for the approval from the FDA for a drug, Drisapersen, that could help ease the pain, and change their child’s life.

Mackenzie Crawford is 15-years-old and has been suffering with the chronic illness since he was 4,

He said that there are many daily tasks that he has to have assistance with.

"I have to get help out of bed, help getting dressed and have somebody help me get my breakfast.

Mackenzie is currently taking Drisapersen and has been for the past 3 years.

However, he is only on a trial basis.

He and others who suffer from Duchenne are looking for the approval from the FDA to regulate the drug.

Todd Crawford, Mackenzie father, spoke before the FDA in late November of this year and gave his testimony about why this drug is so important for Mackenzie and others who suffer from the disease.

"They are heart wrenching milestones like losing his ability to climb stairs, walk long distances, or get himself ready for school. Eventually, he will lose the ability to raise his arms and hug us goodnight."

When Mackenzie broke his leg the Crawford’s said they feared the worst.

"Teenage boys who have Duchenne Muscular Dystrophy are A-typical in regards to regaining they’re ability to walk."

But Mr. Crawford said he credits the drug with assisting in the healing process.

"It is my most sincere belief that Drisapersen preserves his muscle and allowed him to avoid the effects of muscle atrophy and maintain his ability to walk. If approved, this would be one of the biggest drug breakthroughs in about 30 years because there’s never been approved for Duchenne."

Mackenzie and his family said they remain optimistic that the drug will be able to change the lives of all who suffer from Duchenne.

"Our ultimate goal and objective is to kind of eradicate and make sure that all boys are able to have healthy and fulfilled life."

The FDA will announce whether they have approved the drug on December 27th.

If you would like to learn more information about Duchenne click here.