Jul 5, 2023 - Health

Columbus researchers achieve medical breakthrough

Tyler Buchanan

a stethoscope with a glowing lightbulb at the end of it — Illustration: Tiffany Herring/Axios

Breakthrough research at Nationwide Children's Hospital has produced a long-awaited treatment for a rare genetic disorder seen in young kids.

Driving the news: The FDA recently approved Elevidys, the first gene therapy meant to treat Duchenne muscular dystrophy (DMD) in 4- and 5-year-old patients.

Why it matters: Around 20,000 children around the globe are diagnosed with DMD each year.

The genetic condition gradually weakens muscle, potentially leading to fatigue, loss of motor skills, heart problems and breathing issues.

What happened: Developing this treatment has long been a dream of Jerry Mendell, a principal investigator at the Abigail Wexner Research Institute's Center for Gene Therapy who has researched DMD for over 50 years.

"This is what I've devoted my life to," Mendell said in a Nationwide Children's news release.
He co-invented Elevidys alongside Louise Rodino-Klapac, a former Nationwide Children's researcher now with Sarepta Therapeutics.
FDA licensed the treatment to Sarepta, which operates a gene therapy research facility near Easton.

Add Axios on Google

Columbus researchers achieve medical breakthrough

More Columbus stories