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  • Snapshot
    • A 3-year-old girl is brought to the pediatrician by her mother. She had been growing well and meeting all her developmental milestones until a few months ago, when she started displaying hand movements that were unusual. On exam, she is active but frequently wrings her hands. Speech and language testing shows that she has regressed to the level expected of a 2-year-old. She is also noted to avoid any eye contact and showed disinterest in playing. She is sent for genetic testing.
  • Introduction
    • Overview
      • Rett syndrome is a degenerative neurological disorder affecting females, caused by sporadic mutation in MECP2 gene on the X chromosome
    • Pathogenesis
      • characterized by 4 stages
        • stunted development
        • rapid regression of skills
        • pseudostationary
        • late motor deterioration
    • Prognosis
      • decreased life expectancy
  • Presentation
    • Symptoms
      • onset of symptoms typically occur between ages 1-4
      • common symptoms
        • regression of speech and language, motor, and cognitive skills
        • ataxia
        • stunted growth
        • avoidance of eye contact
        • unusual calmness
        • eventually results in cessation of walking
      • gastrointestinal symptoms
        • constipation
        • gastroesophageal reflux
        • difficulty swallowing
    • Physical exam
      • inspection
        • hand-wringing
        • seizure disorder
        • rigidity
        • scoliosis
  • Imaging
    • Radiograph
      • indication
        • evaluate for scoliosis
      • findings
        • scoliosis
    • Barium swallow
      • indication
        • difficulty swallowing
      • findings
        • may have increased tendency for aspiration
  • Studies
    • Genetic testing
      • diagnostic
  • Differential
    • Autism spectrum disorder (ASD)
      • key distinguishing factor
        • patients with ASD typically do not display regression in developed skills
  • Complications
    • Aspiration pneumonia
    • Seizure disorder
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