Spinal Muscular Atrophy Clinic

Unprecedented Focus on SMA Research and Clinical Innovations

SMA Type 1: How Gene Therapy Works

Watch Now

Spinal Muscular Atrophy Clinic

Spinal muscular atrophy (SMA) is the most common form of motor neuron disease. Motor neurons control the voluntary muscles that are used for activities such as crawling, walking, head and neck control, and swallowing. SMA primarily affects infants and children, and it occurs in one in 10,000 births. This makes SMA the most common fatal genetic disease of infants, and the second most common (after cystic fibrosis) autosomal recessive genetic disorder. The carrier rate for this disorder in individuals of European Caucasian descent is one in 40. Thus, it is estimated that there are seven million carriers in the United States.

US News Badge Neurology and Neurosurgery

Neurology & Neurosurgery is ranked by US News & World Report.

Services We Offer 

The SMA Clinic is a joint program between Nationwide Children’s Hospital and The Ohio State University Wexner Medical Center, providing comprehensive, multidisciplinary care for patients from across the United States. Specialized services include the following disciplines:

Spinal Muscular Atrophy Clinic Advocacy

  • We assist patients and families cope with the chronic effects of SMA. We care for infants, children and adults.
  • We assist families identify services available to patients with SMA.

Spinal Muscular Atrophy Clinic Education

  • We provide clinical teaching for medical students, residents, graduate students and post-doctoral fellows.
  • We teach pediatricians, pediatric neurologists and neurologists at local and national conferences.
  • We provide education and educational materials for patients and families.

Research for Spinal Muscular Atrophy

SMA Research News

A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1), according to a recent study led by Dr. Jerry Mendell and published in the New England Journal of Medicine. Information for parents of children with SMA1 who want to learn more about study participation can visit: studysmanow.com

How does Gene Therapy Work?

The NeuroNEXT SMA Biomarker Study

Nationwide Children’s Hospital is a participating clinical trial study site for the 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.

Learn more about spinal muscular atrophy research at Nationwide Children's Hospital.