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Celebrating an Incredible Milestone
On June 22, 2023, in an announcement that resonated across the globe, the U.S. Food and Drug Administration approved the first-ever gene therapy for Duchenne Muscular Dystrophy (DMD).
It was an extraordinary announcement for children ages 4-5 years who live with DMD, as well as an extraordinary achievement for Nationwide Children’s Hospital and for Dr. Jerry Mendell. Over the course of his storied five-decade career as a neurologist and researcher, Dr. Mendell has been devoted to discovering new treatments for children with neuromuscular diseases. He has often said his passion was sparked when he saw his first patient with DMD more than 50 years ago. STAT News called Dr. Mendell “the most famous Duchenne doctor on the planet.”
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It is remarkable for any hospital to have developed an FDA-approved gene therapy, let alone a pediatric hospital. And Nationwide Children’s has now developed two of them.
Dr. Mendell and his colleagues pioneered the first approved gene therapy developed at Nationwide Children’s – Zolgensma, for children with Spinal Muscular Atrophy (SMA), which was called the most important scientific breakthrough of 2017 by the readers of Science magazine.
Now, he and his colleagues have pioneered a second FDA-approved gene therapy. The DMD gene therapy was invented in the Abigail Wexner Research Institute’s Center for Gene Therapy by Dr. Mendell and Dr. Louise Rodino-Klapac. Dr. Rodino-Klapac is now the chief scientific officer at Sarepta Therapeutics, which has licensed the therapy, called ELEVIDYS.
In addition, dozens of Nationwide Children's scientific, administrative, and clinical staff have supported the development and testing of this new treatment over many years. Dr. Kevin Flanigan, director of the center, has been a champion for these breakthroughs.
There is still research we are awaiting to learn the full effects of the gene therapy on children who have DMD. For now, though, we are celebrating this incredible milestone.
DMD is a severe form of muscular dystrophy, typically affecting boys and progressing over time to impact walking, breathing and heart function, resulting in early death. It’s caused by a mutation in a specific gene, and the newly approved therapy delivers a new, functional gene to overcome the effects of the disease.
