A pair of researchers from Binghamton University have received approval from the U.S. Food & Drug Administration for a drug that could treat the world’s most common genetic disease.

The two professors spent the past 13 years developing "Agamree" to treat Duchenne muscular dystrophy, which impacts one in every 5,000 children. Roughly 20,000 are diagnosed with DMD each year.

The most common genetic disease in the world leads to the loss of protein in muscle tissues, which causes progressive weakness and makes completing day-to-day activities challenging.

Professors Eric Hoffman and Kanneboyina Nagaraju are longtime friends who have spent most of their professional careers working to treat the disease.

Agamree is said to be a valuable alternative to corticosteroids, which is the current standard of care in children with DMD. It also doesn’t have the side effects of the other drugs on the market, which can cause bone and growth disturbances.

For the professors, it’s all about helping as many kids as possible.

"First, it's a collaborative one with the families and patients who can't carry out the clinical trials without them. And so this was done in 33 sites in 11 countries. And it really a lot of credit goes to them, the families themselves. And then you can return this to them. And patients lose ambulation and some die due to bone breaks from corticosteroid. So to be able to protect them from that is terrific," said Eric Hoffman, School of Pharmacy professor at Binghamton University.

While the FDA approval is still new, the drug could hit the market as early as the first quarter of 2024.

The two professors say clinical trials are currently underway to treat other forms of muscular dystrophy in adults.