Florian P. Thomas, M.D., Ph.D.
Chair of the Neuroscience Institute and Department of Neurology, Director of the Hereditary Neuropathy Foundation Center of Excellence at Hackensack University Medical Center
Updated: December 10, 2018
Florian P. Thomas, M.D., Ph.D., chair of the Neuroscience Institute and Department of Neurology, and director of the Hereditary Neuropathy Foundation Center of Excellence at Hackensack University Medical Center, is helping to lead an international phase-3 study of a treatment for Charcot-Marie-Tooth (CMT) hereditary neuropathy.
CMT disease is a chronic, 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.
Dr. Thomas, led the U.S. arm of the study which yielded positive results. The trial examined the use of a PXT3003, a new therapy developed by the pharmaceutical company Pharnext, for patients with mild to moderate CMT1A. The study assessed the efficacy and safety of PXT3003 compared to placebo in over 300 participants in Europe, the USA and Canada.
If approved, it could be the first treatment designed to directly affect CMT1A’s disease process.