FDA gives approval for first drug to treat Duchenne muscular dystrophy


INDIANAPOLIS (WISH) — Big news out of the medical industry for those battling Duchenne Muscular Dystrophy.

For the first time, medication could now be available for treatment. On Monday, the FDA gave an accelerated approval to treat the progressive disease.

The move means it can now move towards clinical trials.

The drug is called Eteplirsen and it’s being manufactured by Sarepta Therapeutics out of Massachusetts. The maker says it can slow the progression of DMD, helping them find an underlying cause.

“This is what we’ve been fighting for so we’re ready. We’re excited,” said Laura McLinn, who’s son Jordan has been battling the disease for over 2 years. “We were almost at a point where we were losing hope. Last week I honestly thought that I was losing my mind, I felt like the FDA was going to make me lose my mind.”

We first met Jordan in the winter of 2014. Since then, McLinn has taken Jordan to the statehouse, the Capitol Building, and they even met with Speaker Paul Ryan as they fought for co-called “Right to Try” laws allowing terminally ill patients to use drugs not yet approved by the FDA.

“You have to be proactive because it’s not going to happen without that piece,” said McLinn.

DMD affects about 1 in 3,600 male infants worldwide. The muscle-wasting disease will likely leave them unable to walk by the early teens, and can also lead to heart and respiratory problems. Most patients live into their 20s and 30s.

“It’s a game changer,” said Rob Springer the executive director of the Central Indiana Muscular Dystrophy Association. “For the first time ever an actual treatment that’s moving toward being on the market.”

Springer is fighting this disease professionally and personally.

“Personally as a dad it’s a blessing a curse that it’s a slow progression, but it is a progression. It doesn’t end. And our son Samuel who is 13 years old, he’s at the peak of his life expectancy. The prime of his life right now.” said Springer.

The Central Indiana MDA spent over 60 years raising money for a cure .

As for Jordan and the McLinn family, mom says Monday’s decision impacted everyone.

“All of my kids were very happy. So Jordan might not understand the full depth of what’s happening, but he gets it enough,” said McLinn.

Jordan is in the first grade now. His next step is clinical trials.

McLinn says when they start she will try to get him into one.

It’s important to note that the FDA can revoke the approval at anytime if it doesn’t do what the company says it will do.

The drug is already being used in Europe.



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