Charcot-Marie-Tooth Disease

Neurology / Genetics

Overview

Definition

A heterogeneous group of inherited peripheral neuropathies characterized by progressive distal muscle weakness, muscle wasting, and sensory loss.

Epidemiology

The most common inherited neuropathy, affecting ~1 in 2,500 people.

Etiology & Risk Factors

  • Genetic mutations (most commonly PMP22 gene duplication in CMT1A) affecting myelin or axonal proteins

Clinical Symptoms

  • Foot drop and frequent tripping
  • High-arched feet (pes cavus) and hammer toes
  • 'Stork leg' or 'inverted champagne bottle' leg appearance (distal muscle wasting)
  • Weakness in hands (clumsiness, difficulty writing)
  • Distal sensory impairment

Clinical Approach

Diagnosis

  • Clinical exam
  • Nerve conduction studies (slowed conduction velocity in demyelinating CMT)
  • Genetic testing (definitive)

Management

  • Physical therapy and orthotic devices (AFOs - ankle-foot orthoses)
  • Surgical correction of severe skeletal deformities (pes cavus)
  • Pain management

Complications

  • Scoliosis
  • Severe mobility impairment
  • Recurrent foot injuries and neuropathic ulcers