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Phone: 773-751-9010

E-Mail: smard@smashsmard.org

Address: 3 Londonderry Lane

               Lincolnshire, IL 60069

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smashSMARD is recognized as tax-exempt under section 501(c)3 of the Internal Revenue Code. EIN: 82-5388567

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Gene Therapy Rescues Disease Phenotype in SMARD1 Mouse Model

March 13, 2015

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

Evelyn's Story: Gene Therapy

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.

Ohio Kids' Hospital Quietly Becomes Ground Zero for Gene Therapy

April 15, 2019

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

Gene Therapy Brings Hope to Rare Disease

May 2, 2019

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

FDA Approves Gene Therapy Drug for SMA

May 24, 2019

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

AveXis Presents Zolgensma Data in Broad Spectrum of SMA Patients

May 6, 2019

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

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