Understanding Homocystinuria

A Patient's Guide to Causes, Symptoms, and Treatment

Endocrinology

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Last updated: Mar 24, 2025

What is Homocystinuria?

Homocystinuria is a rare inherited metabolic disorder that affects how the body processes certain amino acids, particularly methionine. The condition results in elevated levels of homocysteine in the blood and urine.

Causes

  • Genetic Inheritance: Inherited in an autosomal recessive pattern
  • Enzyme Deficiency: Most commonly caused by lack of cystathionine beta-synthase (CBS)
  • Metabolic Disruption: Inability to properly process methionine and homocysteine

Common Symptoms

Physical Characteristics

  • Tall, slender build
  • Long limbs
  • High-arched palate
  • Chest deformities

Vision Problems

  • Lens dislocation (ectopia lentis)
  • Severe nearsightedness
  • Glaucoma

Cardiovascular Issues

  • Blood clotting problems
  • Early atherosclerosis
  • Risk of stroke and heart attacks

Skeletal Problems

  • Osteoporosis
  • Scoliosis
  • Long, spider-like fingers

Diagnosis

  • Newborn screening
  • Blood tests for homocysteine levels
  • Genetic testing
  • Urine tests

Treatment Options

Dietary Management

  • Low-methionine diet
  • Protein restrictions
  • Vitamin B6, B12, and folate supplements

Medical Monitoring

  • Regular blood tests
  • Eye examinations
  • Cardiovascular assessments
  • Bone density scans

Living with Homocystinuria

Lifestyle Considerations

  • Regular exercise within safe limits
  • Avoiding blood clot risks
  • Proper dental care

Support Resources

  • Genetic counseling
  • Nutritionist consultation
  • Support groups

Prevention of Complications

  • Early diagnosis and treatment
  • Medication compliance
  • Regular medical check-ups
  • Dietary adherence

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