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
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.
Roach Honored with CNS Hower Award
Award recognizes outstanding teachers and scholars who have generously served the Child Neurology Society.
Childhood Stroke Increases Adaptive and Social Behavior Impairment
After stroke, children who have normal cognition and behavior may still have impaired social adjustment and participation.
Surprising Findings for Exon Skipping in Muscular Dystrophy
Which exons are skipped in therapy for DMD may determine whether significant improvement is made.
Video Game Technology Helps Measure Movement in Patients with Muscular Dystrophy
Researchers hope new technology will expand inclusion criteria for clinical trials to incorporate patients in wheelchairs.
Gene Therapy Trial Improves Walking Performance for Becker Muscular Dystrophy
In a Phase 1/2a trial, BMD patients receiving treatment showed improvements in walking ability over the placebo.
Spotlight: Neurosciences Center
What’s new and what’s ahead?
Alleviating POTS Symptoms with Compression Stockings
Compression of the legs and belly alleviates symptoms of postural orthostatic tachycardia syndrome, a new study suggests.
Studying the Connection between Tourette Syndrome and Sleep Problems
More than 60 percent of pediatric Tourette patients have a sleep problem adding lack of sleep to the list of comorbid conditions.
Dystrophin Isoform Could Lead to DMD Therapy
Studies of a new protein isoform suggest it may offer a novel therapeutic approach for some Duchenne patients.
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.