Jerry Mendell, MD, Announced as King Faisal Prize Laureate in Medicine

Award recognizes Dr. Mendell’s legacy of dedication to treatment for neuromuscular diseases.

January 18, 2024
Jerry  Mendell, MD

Jerry Mendell, MD, senior advisor in the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children’s Hospital, is this year’s recipient of the King Faisal Prize for Medicine.

(COLUMBUS, Ohio) – Jerry Mendell, MD, senior advisor in the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children’s Hospital, is this year’s recipient of the King Faisal Prize for Medicine for his contributions toward the screening, diagnosis and treatment of neuromuscular diseases, including spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD) and limb girdle muscular dystrophy. Dr. Mendell is the first researcher from Nationwide Children’s to be recognized with this award.

“Nationwide Children’s strives to advance treatments from the bench to the bedside every day, and Dr. Mendell’s passion has resulted in exactly that,” said Dennis Durbin, MD, MSCE, president of the Abigail Wexner Research Institute at Nationwide Children’s. “It is thrilling to see him recognized for the work he has done, and more importantly, for the hope he has provided to so many families.”

As an attending neurologist and principal investigator, Dr. Mendell’s career has been dedicated to developing new treatments for devastating neuromuscular diseases since seeing his first patient with DMD more than 50 years ago. Of the first eight gene therapies approved by the Food and Drug Administration (FDA), two have come from Dr. Mendell’s lab.

Dr. Mendell led the Phase 1 clinical trial that was the first to study gene therapy for SMA type 1, the most severe form of SMA. This seminal research performed at Nationwide Children’s led to the development and 2019 FDA approval of Zolgensma® for SMA. He also led the first Phase 1 study of a systemic gene therapy for DMD. This research led to the 2023 FDA approval of ELEVIDYS for DMD.

Dr. Mendell was elected to the National Academy of Medicine in 2021, and he was the first recipient of the American Society for Gene and Cell Therapy (ASGCT)’s Jerry Mendell Translational Medicine Award. He has published more than 400 articles and authored books on skeletal muscle disease, peripheral nerve disorders, and gene therapy.

As an international leader in pediatric research, Nationwide Children’s is actively involved in more than 2,500 clinical research projects to transform health. The Center for Gene Therapy is engaged in a robust pipeline of gene therapies in development for numerous genetic conditions in various stages of research.

Launched by the King Faisal Foundation in 1979, the King Faisal Prize has awarded 295 laureates across five different categories for distinguished contributions to difference sciences and causes. Evaluation by specialized selection committees is based on laureates’ merit and works. The selection process meets international standards.

About The Abigail Wexner Research Institute at Nationwide Children's Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report’s 2022-23 list of “Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit free-standing pediatric health care systems providing unique expertise in pediatric population health, behavioral health, genomics and health equity as the next frontiers in pediatric medicine, leading to best outcomes for the health of the whole child. Integrated clinical and research programs are part of what allows Nationwide Children’s to advance its unique model of care. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s faculty train the next generation of pediatricians, scientists and pediatric specialists. The Abigail Wexner Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational,  behavioral and population health research. The AWRI is comprised of multidisciplinary Centers of Emphasis paired with advanced infrastructure supporting capabilities such as technology commercialization for discoveries; gene- and cell-based therapies; and genome sequencing and analysis. More information is available at NationwideChildrens.org/Research.

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Neuromuscular Disorders