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Treatment of Adrenoleukodystrophy

Treatment for adrenoleukodystrophy involves dietary changes, medications, and potentially stem cell therapy. Early detection is critical to initiating therapies that slow disease progression and improve patient outcomes. Hormonal therapies may also be used to address adrenal insufficiency associated with the condition.

  • 07 Jan 2025
  • 3 min read
  • 20 views

Adrenoleukodystrophy (ALD) is a genetic disorder that affects between one in 14,000 and one in 17,000 male births globally. There is no definitive cure for ALD, which damages the adrenal glands and nervous system. However, early diagnosis and treatment of adrenoleukodystrophy may help combat the condition effectively.

What is the Treatment of Adrenoleukodystrophy?

The treatment of adrenoleukodystrophy aims at reducing the symptoms through medications or surgical procedures. The therapy varies according to the type of ALD.

Treatment options for childhood cerebral adrenoleukodystrophy (cCALD) are:

  • Targeted therapy involves hematopoietic stem cell transplantation (HSCT), which assists in developing blood cells. This therapy effectively cures damage done to the myelin sheath covering the nerve cells.
  • Lorenzo's oil, a mixture of erucic and oleic acid, reduces the progression of ALD.
  • Individualised education plans (IEP) for children with attention-deficit/hyperactivity disorder (ADHD).
  • Speech-language therapy sessions are conducted to improve communication skills.

There is no effective cure for adrenomyeloneuropathy (AMN). Healthcare professionals treat individual symptoms.

Adrenal insufficiency, or Addison’s disease, is cured by hormonal supplements such as corticosteroids. These are synthetic versions of hormones produced by the adrenal glands. Mineralocorticoid replacement therapy prevents sodium loss and reduces increased potassium levels in the blood.

The progression of early cerebral ALD may stop with bone marrow transplantation. The donor's bone marrow contains stem cells that don't have mutations in the ABCD-1 gene. These stem cells replace the patient's defective blood cells and stop brain inflammation. 

Conclusion

Regular magnetic resonance imaging (MRI) brain scans help doctors detect potential damage. Ensure your newborn is screened for ALD right after birth. Having health insurance can substantially reduce the financial burden of treatment of adrenoleukodystrophy.


Disclaimer: The information provided in this blog is for educational and informational purposes only. It is not intended as a substitute for professional advice, diagnosis, or treatment. Please consult your general physician or another certified medical professional for any questions regarding a medical condition. Relying on any information provided in this blog is solely at your own risk, and ICICI Lombard is not responsible for any effects or consequences resulting from the use of the information shared.

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