Spinal Muscular Atrophy Research :: Nationwide Children's Hospital

Research for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is the most common form of motor neuron disease. It is the most common fatal genetic disease of infants, and the second most common (after cystic fibrosis) autosomal recessive genetic disorder. Nationwide Children’s Hospital is currently conducting promising new ground-breaking research to find a cure for Spinal Muscular Atrophy. In 2014, Jerry Mendell, MD began a Phase 1 Gene Transfer Clinical Trial for SMA Type 1. For more information including participation criteria for this clinical trial, click here

The Spinal Muscular Atrophy Clinic at Nationwide Children’s Hospital is the only center in the world that has carried out clinical trials in all three major forms of the disease. Several trials are ongoing and additional trials are in the planning stages.

The NeuroNEXT SMA Biomarker Study

  • Nationwide Children’s Hospital is a participating clinical trial study site for the NeuroNEXT SMA Biomarker Study. View the SMA Biomarker Study video. For questions and more information on this study, or if you wish to participate, please contact Karen Carter, CCRC, Coordinator, Nationwide Children’s Hospital.

Clinical Research Program

  • The Spinal Muscular Atrophy Clinic, along with the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital and the OSU Division of Neuromuscular Medicine, is actively involved in clinical trials for children and adults with SMA. Our Clinical Research Unit is the only center in the world that has carried out clinical trials in all three major forms of the disease. Several trials are ongoing and additional trials are in the planning stages. To that end, we participate in the Families of SMA-sponsored “Project Cure SMA” an international consortium of medical centers performing clinical trials in SMA.

Translational Research Program

  • The translational research program in many ways forms the “soul” of the SMA Clinic. The overarching goal of the program is to use the knowledge gained from research and pre-clinical trials in animals to develop effective therapies for patients with SMA. The SMA Translational Research Program is a joint venture of The Research Institute at Nationwide Children’s Hospital, OSU’s Wexner Medical Center, selected academic medical centers, and pharmaceutical and biotechnology companies.

The SMA Clinic is fully affiliated with the International Coordinating Committee (ICC) for SMA that links the multiple SMA lay and professional organizations into a unified and cohesive unit to pool information and projects related to SMA.

Spinal Muscular Atrophy Research Faculty

Brian K. Kaspar, PhD

featured video

Nationwide Children’s has done more gene therapy clinical trials for neuromuscular disease than any place in the world. Right now, our ground-breaking SMA1 clinical trial is providing hope to Tenley and all families affected by SMA.
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