Coordinated Care From a Range of Specialists, all With Expertise in Pediatric Neuromuscular Disorders
The Center for Gene Therapy | Inside Dr. Jerry Mendell's Research Lab
Groundbreaking. Multidisciplinary. Comprehensive. These words don’t just describe our world-class Neuromuscular Disorders program. They are fundamental to our work and the commitment we make to the families who come to us from across the country. Patients benefit from coordinated care from specialists, all with expertise in pediatric neuromuscular disorders.
Landmark Approval for a Life-Changing Treatment
For almost two years, Nationwide Children’s has reported on the astonishing results of a treatment developed here for a devastating genetic condition. Children with this condition, called spinal muscular atrophy type 1 (SMA1), usually do not have the ability to hold their heads up, to roll over or to sit on their own. Most of these children die before their second birthdays.
The therapy, possible because of decades of research at Nationwide Children’s and The Ohio State University, has allowed a few of these children not only to live, but to crawl, walk and run. Jerry Mendell, MD, a principal investigator in the Center for Gene Therapy in the Abigail Wexner Research Institute, conducted the first experimental trial in children here. The readers of Science magazine named the results of the trial as the most important scientific breakthrough of 2017.
The FDA issued an approval for the gene therapy that has already changed the lives of patients at Nationwide Children’s, laying the groundwork for it to change the lives of children with SMA all over the country. The therapy is called Zolgensma, and a gene therapy company we helped create, AveXis, Inc. (now part of Novartis), is bringing it to market.
When Milan and Elena found out they were pregnant with their second child, Evelyn, they prayed for a healthy baby. Unfortunately, that was not the case. Evelyn tested positive for SMA Type 1. Soon after, Evelyn’s family found out about the clinical trial going on at Nationwide Children’s Hospital. Today, Evelyn is a healthy, normal child. Something we never before have been able to say about a child with SMA Type 1. Now, her parents are thinking long-term and planning for college.
Inside the Center for Gene Therapy at Nationwide Children’s Hospital is where Dr. Mendell has spent nearly 50 years advancing science for neuromuscular disease through clinical trials. It’s where new treatments and potential life-saving cures are being discovered for families who are racing against the clock for options.
The Muscular Dystrophy Clinic is nationally recognized for leading research and clinical care for patients with muscular dystrophy. Supported in part by the Muscular Dystrophy Association, we provide the highest level of comprehensive care to patients from across the country.
Patients from across the country come to Nationwide Children's for the specialized care in our Neuromuscular Disorders Clinic. Our team works closely with the patient and their family to diagnose, treat and manage quality of life.
The SMA Clinic is a joint program between Nationwide Children’s and The Ohio State University Wexner Medical Center. Our team provides comprehensive, multidisciplinary care for patients from across the United States.
Services We Offer
State-of-the-art diagnostic services include:
- Electromyogram (EMG) and nerve conduction testing
- Genetic counseling and genetic testing
- Muscle biopsy
- Nerve biopsy
- Neuromuscular laboratory service
Nationwide Children's world-class Neuromuscular Disorders program, which provides coordinated care with pediatric experts from more than a dozen specialties.
Meet Our Leadership
Kevin M. Flanigan, MD, is an attending neurologist at Nationwide Children's and director of the Center for Gene Therapy in The Research Institute at Nationwide Children's Hospital.
Neuromuscular Disorders research is led by Dr. Kevin Flanigan, a distinguished muscular dystrophy researcher and Director of the Center for Gene Therapy at The Research Institute. Learn more about Dr. Flanigan and his team's neuromuscular research.
Resources for Families
Located across the street from Nationwide Children’s Hospital, families can find a place to call home while their children are being treated at the hospital.
Hosted by Kevin Flanigan, MD, "This Month in Muscular Dystrophy" podcasts highlight the latest in muscular dystrophy and other inherited neuromuscular disease research.
Appointments at our Neuromuscular Disorders Clinics require physician referral. Many insurance plans require authorization from the referring physician in order for the patient's care to be covered. Once all necessary referral and authorization information is received, the patient evaluation will be scheduled.
To schedule a visit, please refer your physician to our Neuromuscular Disorders Clinic using our online Request an Appointment form.
Inside Neuromuscular Disorders
Meet Our Team
Patients benefit from coordinated care from specialists, all with expertise in pediatric neuromuscular disorders.
Conditions and Symptoms
Read our comprehensive list of conditions that we treat and common symptoms of these conditions.
Gene Therapy Clinical Research
Learn more about ongoing clinical trials and other clinical research related to muscular dystrophies and other disorders.