Jerry R. Mendell makes TIME100 Health list for pioneering research

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by Mary Chapman |

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Jerry R. Mendell, MD, has been named to the inaugural TIME100 Health list for his decades-long contributions to the treatment of neuromuscular diseases — such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) — and the advancement of gene therapy.

The global media brand’s list recognizes the impact, innovation, and achievement of 100 individuals who most influenced global health this year. It represents a variety of industries and includes researchers, doctors, educators, advocates, and others who seek to create change for a healthier population. To compile the list, Time and its team of health correspondents and editors spent months consulting experts and sources globally.

“Health innovation, like this list, reflects humanity at its best: people using all their resourcefulness and ingenuity to help one another live better,” Time states.

Mendell, who began his career at the U.S. National Institutes of Health, recently retired from his clinical and research role at Nationwide Children’s Hospital. He now serves as an advisor to the hospital’s Center for Gene Therapy in the Abigail Wexner Research Institute, and to Sarepta Therapeutics.

“Dr. Mendell’s humility and compassion for patients and families shines through his relentless focus on advancing science with the potential to change the course of devastating, fatal and once untreatable neuromuscular diseases,” Louise Rodino-Klapac, PhD, executive vice president, head of research and development, and chief scientific officer at Sarepta, said in a press release.

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Gene therapy pioneer for SMA

Jerry Mendell. Zolgensma

Jerry Mendell, MD, has been recognized by Time magazine as one of the top 100 most influential people in health in 2024. (Photo courtesy of Nationwide Children’s Hospital)

A gene therapy pioneer for SMA, Mendell was a principal investigator across the clinical program for Zolgensma (onasemnogene abeparvovec-xioi), the second SMA treatment to be approved in the U.S. and the first gene therapy for the condition. While at Nationwide Children’s, he served as an investigator for a number of clinical studies, including those for Duchenne and limb-girdle muscular dystrophies.

Mendell spent decades at Nationwide as a clinician and scientist seeking to understand the progressive muscle wasting in DMD and ways to slow or change the disease course. More than a half century after seeing his first DMD patient, Mendell teamed up with Sarepta to develop Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for DMD to be authorized for commercial use. The treatment, which was first investigated at Nationwide Children’s, was granted accelerated approval in the U.S. last June.

Mendell was also a recipient this year of the King Faisal Prize Laureate in Medicine, and in 2021, he was elected to the National Academy of Medicine. Also that year, he became the first recipient of the Jerry Mendell Translational Medicine Award from the American Society for Gene and Cell Therapy. Mendell has published more than 400 peer-reviewed articles and books about gene therapy, skeletal muscle disease, and peripheral nerve conditions.

“I have had the honor of working with Dr. Mendell for many years, first at Nationwide Children’s Hospital and now at Sarepta, to see some of his life’s work come to fruition,” Rodino-Klapac said. “Dr. Mendell’s accomplishments in the field of neuromuscular disease, and the care that he provided to patients and their families in more than 50 years of practice, continue to inspire Sarepta and others working to advance the science of genetic medicine.”

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