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Updated: Jun 16 2021

Rett Syndrome

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https://upload.orthobullets.com/topic/4107/images/rett_xray.jpg
  • Summary
    • Rett Syndrome is a congenital condition caused by an X-linked dominant mutation in the MECP2 gene that affects girls between 6-18 months of age. Patients present with progressive impairment, developmental delays, and spinal abnormalities. 
    • Diagnosis is made primarily based on patient development history and clinical observations.
    • Treatment involves a multidisciplinary approach to address developmental delay, cognitive abnormalities, and orthopedic manifestations. 
  • Epidemiology
    • Incidence
      • 1 in 10,000 births
    • Demographics
      • male fetuses (only 1 X chromosome) do not survive to term
      • female fetuses (1 mutated gene, 1 normal gene) survive and manifest disease
  • Presentation
    • Symptoms
      • normal development for first 6-18 months of life
      • when symptoms present, they range widely from mild to severe including
        • mental slowing (i.e. signs of dementia)
        • abnormal breathing
        • difficulty ambulating
        • seizures
    • Physical exam
      • apraxia
      • abnormal gait
      • scoliosis
      • decreased head circumference
        • usually starts to become evident at 5-6 months
      • poor circulation indicated by cold, blue extremities
  • Studies
    • Diagnosis is based primarily on patient development history and clinical observations
    • Genetic testing
      • may identify genetic mutation responsible for disease, though not in all cases
  • Treatment
    • Nonoperative
      • symptom management
        • there is no specific treatment for Rett's syndrome
        • assistance with feeding and hygeine
        • treat seizures
      • physical therapy
        • indicated to avoid hand contracture
  • Prognosis
    • Half of children unable to walk after the age of ten due to deterioration of motor function
      • development is normal until 6 to 18 months then progressive impairment and development delays occur
      • regression is rapid until the age of three
      • a more stable phase of progression occurs until age of 10
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