From animal models to patients: new therapies for Charcot-Marie-Tooth disease (CMT)

Charcot-Marie-Tooth disease (CMT) is the most common hereditary neuropathy of the peripheral nervous system. So far no treatment is available. Using transgenic animal models, we have developed new therapeutic approaches which are currently being translated to humans, thus creating new therapeutic options for approximately 1.5 million affected CMT patients worldwide.