DISEASE SCANNER

Global Incurable Diseases Tracker

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Genetic Disorder

Smith-Magenis Syndrome

HIGH SEVERITY

A microdeletion syndrome caused by 17p11.2 deletion including RAI1 gene. Characterized by intellectual disability, behavioral problems, sleep disturbances (inverted circadian rhythm), and self-injurious behavior.

Global Affected

15.0K

Countries

15

Symptoms

Intellectual disability
Behavioral problems
Sleep disturbances
Self-injurious behavior
Speech delay
Distinctive facial features
Hearing loss
Scoliosis

Treatment Options

Genetic counseling
Supportive care
Symptom management
Enzyme replacement (if applicable)
Physical therapy
Regular monitoring
Multidisciplinary care

Risk Factors

1Family history
2Genetic mutations
3Consanguinity
4Advanced paternal age
5Ethnic predisposition

Diagnostic Methods

  • 1Genetic testing
  • 2Clinical evaluation
  • 3Family history assessment
  • 4Specialized laboratory tests
  • 5Imaging studies
  • 6Biopsy (if applicable)

Prognosis

Good with behavioral and medical management. Self-injurious behaviors manageable. Sleep disturbance improves with melatonin. Normal life expectancy.

Prevention

  • Genetic counseling
  • Carrier screening
  • Prenatal diagnosis
  • Preimplantation genetic diagnosis
  • Family planning

Research Status

Melatonin for sleep disturbance. Behavioral interventions. Speech and occupational therapy. Avoidance of medications that worsen behavior. Multidisciplinary management. No cure available.

Sources

  • https://www.ncbi.nlm.nih.gov/books/NBK1116
  • https://medlineplus.gov/genetics
  • https://rarediseases.org

Medical Disclaimer

This information is for educational purposes only. Always consult healthcare professionals for medical advice, diagnosis, and treatment.