Spinal Muscular Atrophy Clinic :: Nationwide Children's Hospital

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.

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 to 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

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.

Physician Referral Information

The Spinal Muscular Atrophy (SMA) Clinic accepts referrals from primary care physicians and pediatric specialists from the United States and internationally. To make a referral, there are three options:

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.

Refer a Patient

Neurology accepts referrals from U.S. and international primary care physicians and pediatric specialists.

Refer by Phone: (614) 722-6200 or 1-877-722-6220

Refer by Fax: (614) 722-4000

Refer a Patient Online »


We have compiled a list of Neuromuscular Disorders resources including:

  • Clinical Trials
  • Family Services
  • Organizations

View our resources page »

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