From studies at the molecular level to the behavioral level, Neurosciences faculty members are investigating how neurological and neuromuscular disorders and conditions develop and working toward new treatment and prevention options.
Faculty members are developing models of neuromuscular disorders, identifying gene-delivery strategies that cross the blood-brain-barrier and lead to clinical trials, including the first human gene therapy trial for Duchenne muscular dystrophy, phase I clinical trial to target limb-girdle muscular dystrophy type 2D and have led the first national muscular dystrophy newborn screening study.
Dr. Jerry Mendell, a distinguished muscular dystrophy researcher, leads the Neuromuscular Disorders clinical program and is the Director of the Center for Gene Therapy at The Research Institute. Dr. Mendell was among the first to test muscle cell transplantation for Duchenne muscular dystrophy in the early 1990’s and the first to study viral mediated gene therapy for muscular dystrophy in humans. He has made fundamental contributions in clinical research and in the molecular genetics of neuromuscular disease. In 2006, he and his team began the first-ever gene therapy trials for boys with Duchenne muscular dystrophy.
The Muscular Dystrophy Association named Nationwide Children’s Hospital to its Clinical Research Network in 2008. In 2010, we received a $7 million National Institutes of Health grant along with designation as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center. The Wellstone Center explores and refines therapeutic strategies that have shown promise in animal models of muscular dystrophy, and ongoing studies include further research in the laboratory as well as clinical trials to assess efficacy in patients. The neuromuscular translational research team includes Dr. Kevin Flanigan and Dr. Zarife Sahenk, investigators at the Center for Gene Therapy.
The SMA Clinic is directed by Dr. John Kissel of the Division of Neuromuscular Medicine at the Ohio State University College of Medicine. In collaboration with the Center for Gene Therapy and OSU, the clinic 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. We have a number of ongoing trials, and many more in planning stages.
We participate in the Families of SMA-sponsored "Project Cure SMA," an international consortium of medical centers performing clinical trials in SMA. We are also part of a select group of 25 national clinical sites that make up Network of Excellence in Neuroscience Clinical Trials (NeuroNEXT). Created by the NIH, NeuroNEXT fosters cooperation among leading clinical sites, allowing them to efficiently design and implement clinical trials encompassing the entire spectrum of neurologic diseases.
Clinical researchers at Nationwide Children's Hospital are committed to identifying new approaches for the prevention, diagnosis and treatment of childhood diseases, taking research discoveries from the lab to the patient's bedside.
Investigators with the Center for Gene Therapy conduct numerous neuromuscular clinical trials including muscular dystrophy and spinal muscular atrophy. To learn about enrolling in one, you may call one of our study coordinators at (614) 722-2203. Information about ongoing clinical trials can also be found at www.ClinicalTrials.gov, a service of the U.S. National Institutes.
Browse additional Neurosciences research studies being conducted at Nationwide Children’s Hospital.
Duchenne Muscular Dystrophy
Spinal Muscular Atrophy
Traumatic Brain Injury/Concussion
Additional Neuromuscular Disorders
The Heart in Neuromuscular Disease
Wellstone Muscular Dystrophy Cooperative Research Center
A Wearable App Helps Track Seizures for Researchers and Families
A partnership between pediatric neurology, research application developers and the world’s largest seizure-tracking database has resulted in a tool to benefit patients, researchers and clinicians.
Tuberous Sclerosis Insights In Depth
Learn about Dr. Steve Roach’s 2015 Hower Award lecture and download a print version.
Gene Therapy for Sanfilippo Syndrome Shows Promise, Safety and Efficacy
An interdisciplinary translational research team is working to bring the therapy to clinical trials.
Batten Disease Clinicians: Ask Families About Sleep Disturbances
Sleep abnormalities are pervasive in patients with neuronal ceroid lipofuscinosis, and targeted treatment may improve quality of life.
Predicting the Duration of Post-Concussion Symptoms
Specific factors lead to prolonged symptoms, and that knowledge may guide further research, concussion management and family expectations.
Searching for a Pediatric Ischemic Stroke Screening Tool
An adult stroke scale could not reliably distinguish between children with acute ischemic stroke and children with focal neurological deficits due to other causes, but this incompatibility provides insight into potential elements of a pediatric tool.
Diagnosing Autoimmune Encephalitis in Children
Researchers identify common patterns in MRI and EEG results which support the diagnosis.
Insights on Neurocutaneous Syndromes
New handbook looks at big picture for these neurological disorders.
Chiari I Malformations: Advances and Challenges to Care
New publication provides comprehensive look at CM-1 diagnosis, treatment and research.
Spotlight: The Neurosciences Center
What’s new and what’s ahead?
Infantile Spasms Respond Poorly to a Common First-Line Treatment
Researchers recommend other initial treatments after finding that topiramate has a low rate of infantile spasms remission
Novel Technique Restores Gene and Function in Dysferlinopathy Animal Models
Clever cutting and delivery allow cell's own mechanisms to recombine needed DNA.
Wellstone Center Designation
Muscular Dystrophy Association's Clinical Research Network
Nationwide Children’s Hospital is one of five centers to be part of the Muscular Dystrophy Association's Clinical Research Network to support trials and studies in Duchenne muscular dystrophy (DMD).
Duchenne Muscular Dystrophy Affects Two Brothers
Jenn McNary's sons both have Duchenne muscular dystrophy. One is receiving experimental therapy and the other is not.