DISEASE SCANNER

Global Incurable Diseases Tracker

Back to Globe
Genetic Disorder

Marfan Syndrome

MODERATE

An autosomal dominant connective tissue disorder caused by FBN1 gene mutation affecting fibrillin-1 protein. Affects skeletal, ocular, and cardiovascular systems. Most serious complication is aortic root dilation/dissection. Equal prevalence across ethnicities.

Global Affected

60.0K

Countries

15

Symptoms

Tall stature with long limbs (dolichostenomelia)
Arachnodactyly
Pectus excavatum/carinatum
Ectopia lentis (lens dislocation)
Aortic root dilation
Mitral valve prolapse

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

Life expectancy near normal with modern care (70-80 years). Aortic dissection main risk, now rare with prophylactic surgery. Without treatment: median survival 32 years. Regular imaging essential.

Prevention

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

Research Status

ARBs (losartan) and beta-blockers slow aortic growth. Aortic root replacement when diameter >5.0 cm (or lower with risk factors). Regular echocardiographic surveillance essential. Gene therapy research early stage.

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.