Childhood-Onset Cerebral X-Linked Adrenoleukodystrophy

Common Name(s)

Childhood-Onset Cerebral X-Linked Adrenoleukodystrophy

X-linked adrenoleukodystrophy (X-ALD) is an inherited disorder that occurs most often in males. It mainly affects the nervous system and the adrenal glands (small glands located on top of each kidney). People with this disorder often have progressive destruction of the fatty covering (myelin) that insulates nerves in the brain and spinal cord. They may also have a shortage of certain hormones caused by damage to the outer layer of the adrenal glands (adrenal cortex), called adrenocortical insufficiency. X-ALD is caused by mutations in the ABCD1 gene. There are three distinct types of X-ALD: a childhood cerebral form, an adrenomyeloneuropathy type, and a type called Addison disease. A specific treatment for X-ALD is not currently available, but eating a diet low in very-long-chain fatty acids and taking special oils can lower the blood levels of very-long-chain fatty acids. Bone marrow transplantation (BMT) is an option for boys and adolescents in early stages who have evidence of brain involvement on MRI.   Gene therapy is being investigated as a treatment for X-ALD; promising results for two young boys who received gene therapy treatement are discussed by Dr. Nathalie Cartier, et. al. in the journal Science.
 

Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Childhood-Onset Cerebral X-Linked Adrenoleukodystrophy" for support, advocacy or research.

ALD Life

A charity to promote awareness, research and prevention of Adrenoleukodystrophy (ALD) and Adrenomyeloneuropathy (AMN). ALD Life enables sufferers of ALD and AMN worldwide a forum to come together to support and advise each other. We provide financial and emotional support as well as practical advice and updates on research innovations, as well as raising funds for research and treatments.

Last Updated: 9 Apr 2014

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Fight ALD

Our mission is to save the lives of our boys by bringing about awareness through education--to our community and medical professionals--about the early onset symptoms of X-ALD (Adrenoleukodystrophy), the simple diagnostic blood test, and treatments. We also have information about the adult onset, AMN (Adrenomyeloneuropathy).

Last Updated: 4 Oct 2012

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General Support Organizations

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How do you compare to others with this condition?

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Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Childhood-Onset Cerebral X-Linked Adrenoleukodystrophy" for support, advocacy or research.

ALD Life

A charity to promote awareness, research and prevention of Adrenoleukodystrophy (ALD) and Adrenomyeloneuropathy (AMN). ALD Life enables sufferers of ALD and AMN worldwide a forum to come together to support and advise each other. We provide financial and emotional support as well as practical advice and updates on research innovations, as well as raising funds for research and treatments.

http://www.aldlife.org

Last Updated: 9 Apr 2014

View Details
Fight ALD

Our mission is to save the lives of our boys by bringing about awareness through education--to our community and medical professionals--about the early onset symptoms of X-ALD (Adrenoleukodystrophy), the simple diagnostic blood test, and treatments. We also have information about the adult onset, AMN (Adrenomyeloneuropathy).

http://www.fightald.org

Last Updated: 4 Oct 2012

View Details

 

General Support Organizations

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General Resources

ALD-AMN Route Map

A dedicated website for providing patients and families affected by Adrenoleukodystrophy (ALD) and Adrenomyeloneuropathy (AMN) a journey of their health and social care needs.

Uploaded By: ALD Life

Updated 4 Apr 2013

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Scientific Literature

Articles from the PubMed Database

Research articles describe the outcome of a single study. They are the published results of original research.
The terms "Childhood-Onset Cerebral X-Linked Adrenoleukodystrophy" returned 0 free, full-text research articles on human participants.

Reviews from the PubMed Database

Review articles summarize what is currently known about a disease. They discuss research previously published by others.
The terms "Childhood-Onset Cerebral X-Linked Adrenoleukodystrophy" returned 0 free, full-text review articles on human participants.

 
 
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Symptoms, Diagnosis, and Treatment

There are currently no related results available in Genetics Home Reference.

There are currently no related results available in GeneReviews.

 
 
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Clinical Trial Information This information is provided by ClinicalTrials.gov

There are currently no open clinical trials for this condition.