Adrenoleukodystrophy (ALD)



Adrenoleukodystrophy (ALD), sometimes called X-linked adrenoleukodystrophy, is a rare but deadly genetic disease affecting approximately one out of 18,000 people. ALD, most severely affecting boys and men, is characterized by progressive neurologic deterioration due to the breakdown of the myelin covering of the brain cells. As the sheath is removed, the neurons cannot tell the muscles and other elements of the central nervous system what to do.

The childhood form (the most severe) sets in between the ages of four and ten, usually causing behavioral issues, withdrawal, speech and hearing issues, loss of concentration, seizures, and hyperactivity. For the childhood form, stem cell transplant and gene therapy are treatment options if the disease is detected early.

 

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