March 13, 2015

Gene Therapy Rescues Disease Phenotype in SMARD1 Mouse Model

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

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.

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 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.

May 24, 2019

FDA Approves Gene Therapy Drug for SMA

The drug, Zolgensma, is being called a "miracle drug" and will be used to treat children with spinal muscular atrophy, or SMA.

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.

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E-Mail: smard@smashsmard.org

Address: 6 Exmoor Lane

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