An update on clinical trials in Charcot-Marie-Tooth Disease

Author(s): Davide Pareyson & Giuseppe Piscosquito

How far away is an effective therapy for Charcot-Marie-Tooth disease (CMT), the most frequent inherited neuropathy affecting approximately one individual in every 2500 around the world [1]? The next few years will be fundamental to giving the answer to this key question about a still untreatable disorder, for which rehabilitation therapy and surgical correction of skeletal deformities still represent the only available approaches. Different lines of research are rapidly providing encouraging data on possible effective treatments in cellular and animal models, and clinical studies are progressively filling the gaps to be fully ready for clinical trials, creating great hope in patients and researchers alike. However, CMT poses a series of difficulties in the pathway to the cure and in running clinical trials, as testified to by the failure of recent randomized-controlled trials (RCT) with ascorbic acid.