Hereditary Sensory Motor Neuropathy (Charcot-Marie-Tooth Disease)
Inherited disease that affects the peripheral motor and sensory nerves
Aetiology
AD, AR, X-linked - hundreds of mutations, demyelinating and axonal varieties
HSMN Ia is the most common type (70%) - autosomal dominant demyelinating neuropathy
Clinical presentation
Symptoms
Typical presenting symptom is weakness of the feet and ankle
Signs
Depressed or absent tendon reflexes with a weakness of foot dorsiflexion at the ankle
An affected adult typically has a bilateral foot drop, symmetrical atrophy of muscles below the knee (stork leg appearance), pes cavus, atrophy of intrinsic hand muscles, especially the thenar muscles of the thumb, and absent tendon reflexes in both upper and lower extremities
Investigations
Tests to exclude other causes of neuropathy e.g. bloods, serum and urine protein electrophoresis, muscle biopsy, CSF
Genetic studies
Nerve conduction studies
Management
Currently there are no effective treatments to reverse or slow the underlying disease process
Management is supportive with input from various members of the MDT