Effectiveness of exercise therapy for individuals diagnosed with Charcot-Marie-Tooth disease: A systematic review of randomized clinical trials.

Charcot-Marie-Tooth disease is a genetic nerve condition that weakens muscles over time, and current treatments like orthoses, surgery and physiotherapy mostly manage symptoms rather than change the disease course. This review asked what randomized trials actually show about exercise therapy for strength, physical function, aerobic capacity and quality of life in people with the condition. The authors pooled six trials covering 214 participants, most diagnosed with the CMT1A subtype, and most trials carried a high risk of bias. The one finding rated as moderate-quality evidence was that strengthening the ankle dorsiflexor muscles appeared to limit worsening weakness over two years in children with CMT1A.

Everything else, including effects on function, aerobic fitness and quality of life, rested on low or very low certainty evidence, meaning confidence in those results is limited and more robust trials are still needed before firmer conclusions can be drawn.

If you or someone you care for has Charcot-Marie-Tooth disease, you may already know that treatments mostly aim to ease symptoms rather than reverse the underlying nerve problem. This review looked at what research evidence actually exists for exercise programs, pulling together results from six small clinical trials involving 214 people in total. The clearest finding came from strengthening exercises targeting the ankle muscles that lift the foot, which seemed to slow the worsening of weakness over two years in children with this condition.

For other outcomes, like overall physical function, stamina during activity, and quality of life, the evidence was much weaker and doesn't allow confident conclusions either way. It's also worth knowing that most of the included trials had a high risk of bias, meaning their results should be interpreted cautiously. This doesn't mean exercise doesn't help, but rather that the research base is still too small and inconsistent to say much with confidence beyond that one ankle-strengthening finding in children.

This is a summary of research, not medical advice. Talk to your own healthcare provider about anything affecting your care.

For clinicians advising patients with Charcot-Marie-Tooth disease on exercise prescription, this review clarifies just how thin the randomized trial evidence currently is. Searching ten databases through July 2021, the authors identified only six eligible trials (214 participants pooled, mean age 38.5 years, 83% with CMT1A), with a mean PEDro score of 5.25 and six of the trials judged high risk of bias. The one result graded as moderate-certainty evidence was that ankle dorsiflexor strengthening reduced progression of weakness at 24 months specifically in children with CMT1A, a finding relevant to pediatric practice but not generalizable across adult populations or other muscle groups without further data.

All other outcomes assessed, including broader function, aerobic capacity, and quality of life, were supported only by low to very low certainty evidence, reflecting small trial sizes, methodological limitations, and inconsistency across studies rather than a demonstrated absence of effect. The review does not establish that exercise therapy broadly improves outcomes in CMT, nor does it compare specific exercise types against each other with confidence. It identifies where evidence is currently strongest, narrowly, in pediatric ankle strengthening, while leaving the effectiveness of exercise for adults and for most other outcomes largely unresolved pending higher-quality trials.