A pair of researchers at Binghamton University received approval from the U.S. Food and Drug Administration for a drug that could treat the world’s most common genetic disease. The two professors worked over the last 15 years developing Agamree, which is set to treat Duchenne muscular dystrophy, a disease that mostly impacts young boys.

One man who has lived with Becker's muscular dystrophy for over 40 years says even if the drug won't help him, he’s pushing to see it help as many kids as possible. While Agamree is set to treat Duchenne, trials are in the works to see if it could treat other forms as well.

Live hasn’t always been easy for Mike Ziemba.

Becker's muscular dystrophy starts off less severe, but gradually makes muscles weaker and smaller. He now spends most of his life in a motorized wheelchair. 

"It's a miserable disease that just kind of presents misery at different levels," he said. "And it's just it's very hard to live with."

But that hasn’t stopped Ziemba from living his life. During high school, he was captain of the marching band, and played golf and basketball. A few years back, he received a Ph.D. from a pharmacy school with one goal in mind.

"If I can be at all involved and part of what gets rid of this terrible disease forever, then I have made a very positive contribution here on Earth,” Ziemba said.

Thanks to his lead professor at Binghamton, Ziemba may just be playing a part in that lifelong goal. Professor Eric Hoffman and his partner at BU received FDA approval for a drug to treat Duchenne muscular dystrophy, the world’s most common genetic disease. 

"It's wonderful for the families, for everybody that everything you've worked on has come to fruition,” said Hoffman, School of Pharmacy professor at Binghamton University.

DMD, as it’s often called, impacts 1 in every 5,000 children, mostly young boys. The only treatments before this drug were corticosteroids, which can have deadly side effects.

"Patients lose ambulation and some die due to bone breaks from corticosteroids. So to be able to protect them from that is terrific,” Hoffman said.

Agamree is a drug that’s said to be a valuable alternative. For Ziemba, the FDA approval is a day he thought he’d never see. 

“I was diagnosed with this disease 40 years ago. And I've been hearing about treatments and such for most of that time. And then a decade ago, I got involved with the exact point that you just made, that I know that the work I'm doing may not help me, but it is going to be beneficial for thousands and thousands of people," he said.

Hoffman said the drug could hit the market in the first quarter of 2024.