That ultra-rare disease is SMARD.

First-time gene therapy, developed at Nationwide Children's, is allowing kids like Evelyn to have a bright future. Watch her incredible story.

AAV9-IGHMBP2 rescued motor function, neuromuscular physiology, and life span (450% increase).

Boys with a rare muscle disease called myotubular myopathy are breathing on their own, thanks to gene therapy.

The biomedical world, fixated on gene therapy now, is coming to Nationwide Children's Hospital in Columbus.

Gene therapy trial data was presented during the 2019 American Academy of Neurology Annual Meeting.