CMT, also known as HMSN, is a hereditary, slowly progressive peripheral neuromuscular disease that often manifests already in childhood. The common clinical features are progressive muscle wasting and weakness together with sensory loss starting in the feet and progressing more proximally including to the hands. Foot deformity is common. The gait becomes clumsy, and twisting an ankle and stumbling are typical even in children and adolescents. Muscle pain, fatigue and increasing loss of mobility are common. Over time, the need for walking aids, walkers, and sometimes wheelchairs follow.  Many doctors are not familiar with the disease, its typical symptoms and neurological causes – this often leads to considerable delay in diagnosis, if diagnosis happens at all. There is still no effective therapy or cure for CMT but there are many research efforts ongoing. However, obstacles to developing therapies include a lack of awareness of the disease, a lack of financial support, the low profitability prospects for industry and the unavailability of sufficient data for researchers. This is typical for the approximately 8000 other rare diseases.