Nationwide Children’s Becomes Qualified Treatment Center for Glycogen Storage Disease Type 1A Gene Therapy

(COLUMBUS, Ohio) - Nationwide Children's has been designated a qualified treatment center for GENGLYCOS™ (pariglasgene brecaparvovec-opnr), a gene therapy for the treatment of glycogen storage disease type 1A (GSD Type 1A).

In August 2026, the U.S. Food and Drug Administration approved GENGLYCOS, which was developed by pharmaceutical company Ultragenyx, for the treatment of GSD Type 1A in adult and pediatric patients 8 years of age and older.

With Ultragenyx's designation, Nationwide Children's becomes part of a network across the U.S. that is certified to have the advanced facilities and specially trained staff necessary for the administration of GENGLYCOS.

"We hope this new treatment leads to improvements in patients' care and quality of life," said Will Burns, MD, pediatric geneticist at Nationwide Children's. "This approval is another major step toward continuing to treat a variety of rare diseases with genetic therapies."

Caused by a defective gene coding for the enzyme G6Pase-α GSD Type 1A is a metabolic disorder that leads to patients' inability to maintain normal glucose levels, causing the accumulation of glycogen in the liver and kidneys. Patients with GSD Type 1A can become severely hypoglycemic, which can be life-threatening.

GENGLYCOS is an AAV8 gene therapy delivered via single intravenous infusion, designed to provide stable expression and activity of the enzyme G6Pase-α.

About Nationwide Children's Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report's 2025-26 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, as well as prioritizing quality and safety, are part of what allows Nationwide Children's to advance its unique model of care. Nationwide Children's has a staff of more than 17,000 that provides state-of-the-art wellness, preventive and rehabilitative care and diagnostic treatment during more than 1.9 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children's physicians train the next generation of pediatricians 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. More information is available at NationwideChildrens.org.

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