October marks CMT Awareness Month, shining a light on Charcot-Marie-Tooth disease (CMT), a group of inherited conditions affecting the peripheral nerves and often causing muscle weakness, sensory changes and difficulties with movement, particularly in the hands and feet.
At TREAT-NMD, we are committed to supporting progress in the diagnosis, care and treatment of people living with neuromuscular diseases, including CMT. This month, we are highlighting the work of CMT UK and the wider CMT community, while recognising important developments in research and clinical trial readiness.
A significant step forward came in August 2026, when organisations representing people affected by CMT, clinicians and industry partners published the first consensus framework for designing CMT clinical trials. Developed through the ToPIC: CMT collaboration, the recommendations aim to provide researchers and pharmaceutical companies with a clearer and more consistent approach to developing potential treatments.
The framework includes recommendations around reducing placebo exposure, using clinical presentation alongside genetic information when appropriate, and identifying meaningful function-based and patient-reported outcomes. It also highlights the potential role of biomarkers and the importance of studying treatments across different stages of life.
These developments are an important step towards making future CMT trials more efficient, inclusive and meaningful for people living with the condition. While there are currently no approved treatments for CMT, greater collaboration across patients, clinicians, researchers and industry is helping to strengthen the pathway towards new therapies.
Throughout October, CMT Awareness Month provides an opportunity to learn more about CMT, share reliable information and support the organisations working to improve lives and advance research.
Find out more about CMT and the latest developments in research through CMT UK for resources and to connect for updates information and support if you are based in the UK and CMTA Â if you are in the USA.
