(COLUMBUS, Ohio) - The Charcot-Marie-Tooth Association has named Nationwide Children’s Hospital a CMT Center of Excellence, in recognition of the comprehensive care and expertise provided to children with the most common form of Charcot-Marie-Tooth disease, CMT1A.
Also called hereditary motor and sensory neuropathy, Charcot-Marie-Tooth disease (CMT) is a group of inherited neurological disorders affecting an estimated 2.8 million adults and children worldwide.
Gene mutations reduce the ability of peripheral nerves to carry motor and sensory information, typically resulting in pain and slowly progressing weakness and muscle atrophy in the feet and lower legs then the hands and arms. In rare cases, CMT can affect breathing. There is currently no treatment for the CMT1A form of the disease.
The hospital joins The Ohio State University and 24 other institutions as a CMT Center of Excellence.
“This puts Columbus Ohio on the map for CMT expertise, with OSU delivering care to adults and Nationwide Children’s delivering care to children,” says Zarife Sahenk, MD, PhD, an attending neurologist and director of Clinical and Experimental Neuromuscular Pathology at Nationwide Children’s. “I am very excited that the CMT Association reached out to recognize our center with others as a Center of Excellence.”
Referrals of children with CMT to the Nationwide Children’s program have grown in recent years, after the hospital began offering a weekly CMT clinic. Nationwide Children’s provides CMT patients physical medicine, orthopedics and physical therapy, genetic counseling, occupational therapy and other services.
The designation as a center of excellence will give CMT patients access to resources provided by CMTA, including educational resources for them and their families, options to attend summer camps and more, says Dr. Sahenk who is a professor of Pediatrics, Pathology and Neurology at The Ohio State University College of Medicine. In addition to sponsoring Centers of Excellence, CMTA also supports research, including a database of 7,000 de-identified patient records used to establish an evaluation scale for children.
Nationwide Children’s will begin a National Institutes of Health-funded clinical trial of a gene therapy designed to protect and restore peripheral nerve function in children and adults with CMT1A later this year.