June 9, 2021
That ultra-rare disease is SMARD.
May 24, 2019
First-time gene therapy, developed at Nationwide Children's, is allowing kids like Evelyn to have a bright future. Watch her incredible story.
March 13, 2015
AAV9-IGHMBP2 rescued motor function, neuromuscular physiology, and life span (450% increase).
May 2, 2019
Boys with a rare muscle disease called myotubular myopathy are breathing on their own, thanks to gene therapy.
April 15, 2019
The biomedical world, fixated on gene therapy now, is coming to Nationwide Children's Hospital in Columbus.
May 6, 2019
Gene therapy trial data was presented during the 2019 American Academy of Neurology Annual Meeting.