DISEASE SCANNER

Global Incurable Diseases Tracker

Back to Globe
Genetic Disorder

Alexander Disease

HIGH SEVERITY

A rare, usually fatal neurological disorder causing destruction of white matter in the brain. Characterized by abnormal protein (GFAP) deposits called Rosenthal fibers. Most common form is infantile, but juvenile and adult forms exist. Affects approximately 1 in 2.7 million people. No cure available.

Global Affected

30.0K

Countries

30

Symptoms

Developmental delay (infantile)
Megaloencephaly (enlarged head)
Seizures
Spasticity
Failure to thrive
Hydrocephalus
Vomiting
Bulbar signs (adult)
Ataxia (adult)
Palatal myoclonus (adult)
Dysarthria
Dysphagia
Progressive dementia

Treatment Options

Antiepileptic medications
Gastrostomy tube
Physical therapy
Occupational therapy
Speech therapy
Respiratory support
Palliative care
Nutritional support
Seizure management
Spasticity management
Research protocols

Risk Factors

1GFAP gene mutation
2Autosomal dominant inheritance
3Most cases spontaneous
4No gender predilection
5No racial predilection
6Genetic (not environmental)

Diagnostic Methods

  • 1MRI of brain (characteristic pattern)
  • 2GFAP genetic testing
  • 3Brain biopsy (rarely needed now)
  • 4Clinical evaluation
  • 5Neuroimaging
  • 6Genetic counseling

Prognosis

Poor. Infantile form fatal within 10 years usually. Juvenile form variable. Adult form slower progression but progressive. No cure available. Life expectancy reduced in all forms. Quality of life significantly affected. Death usually from respiratory or feeding complications.

Prevention

  • Genetic counseling
  • Prenatal testing available
  • Preimplantation genetic diagnosis
  • Carrier screening
  • Family planning support

Research Status

No cure. Supportive care only. Treatment of seizures. Gastrostomy for feeding. Physical therapy. Antisense therapy research ongoing. Gene therapy being investigated. Bone marrow transplant not effective. Palliative care important. Multidisciplinary support essential.

Sources

  • https://www.mayoclinic.org/diseases-conditions/alexander-disease
  • https://www.hematology.org/education/patients
  • https://www.ncbi.nlm.nih.gov/books
  • https://rarediseases.org/rare-diseases

Medical Disclaimer

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