June 9, 2021

Gene therapy biotech is bursting onto the scene with 12 programs in tow. An ultra-rare neuro disease is up first

That ultra-rare disease is SMARD.

May 24, 2019

Evelyn's Story: Gene Therapy

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

F2.large.jpg

March 13, 2015

Gene Therapy Rescues Disease Phenotype in SMARD1 Mouse Model

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

myotubular.jpg

May 2, 2019

Gene Therapy Brings Hope to Rare Disease

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

nationwide.jpeg

April 15, 2019

Ohio Kids' Hospital Quietly Becomes Ground Zero for Gene Therapy

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

May 6, 2019

AveXis Presents Zolgensma Data in Broad Spectrum of SMA Patients

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