Physician at Hackensack University Medical Center Leads First U.S. Clinical Trial of Promising New Drug to Treat Charcot-Marie-Tooth Disease

Hackensack University Medical Center recently announced that an international phase 3 study of a novel treatment for Charcot-Marie-Tooth (CMT) hereditary neuropathy, which in the United States was led by Florian P. Thomas, M.D., Ph.D, chair of the Neuroscience Institute and the Department of Neurology, and director of the Hereditary Neuropathy Foundation Center of Excellence at Hackensack University Medical Center, yielded positive results. CMT disease is a chronic, often severe and debilitating inherited condition affecting some 125,000 people in the United States, of which approximately 50 percent have a particular subtype called Charcot-Marie-Tooth type 1A disease (CMT1A). It causes progressive weakness and impairs sensation resulting, among many other symptoms, in weakness, joint deformities and difficulty walking.