Charcot-Marie-Tooth disease – an inherited neurological disorder
Estimates suggest around 25,000 people in the UK have Charcot-Marie-Tooth disease (CMT).1 They usually start to have symptoms – which can include muscle weakness and numbness in the feet, arms and hands – during childhood or adolescence. Symptoms slowly get worse and more disabling over time, making everyday tasks increasingly difficult. Professor David Bennett, of the University of Oxford, is in the early stages of developing a new treatment – a gene therapy – for children with CMT. There’s no cure for CMT, and no way to slow down the progression of the disease, so improvements in treatment are badly needed.
How are children’s lives affected now?
People with CMT, an inherited condition, usually experience their first symptoms when they’re between five and 15 years old.
Symptoms vary from one child to another. Early signs of CMT in a young child can be difficult to spot. A child might just seem clumsy or accident prone, for example, or they might find it difficult to walk, because of problems lifting their feet off the ground. As the children can have high insteps, it may be difficult to get shoes to fit.
Symptoms get worse and more disabling over time, although CMT is unpredictable, so it’s not possible to tell how quickly this will happen or how severe symptoms will become.