Leukodystrophy

Leukodystrophy is a rare inherited disease that damages the covering (the myelin) of the nerve cells in the brain and spinal column. As that damage occurs, the cells can no longer transmit signals between the brain and the body. This impairs movement, speaking, vision, hearing, and mental development.

Leukodystrophies are actually a group of disorders, including Krabbe disease, metachromatic leukodystrophy, adrenoleukodystrophy, Alexander disease, Canavan disease, Pelizaeus-Merzbacher disease, and vanishing white matter disease. Most of which are genetic. They can be hard to detect early because children seem healthy at first. Symptoms, however, gradually worsen.

There are no cures for any of the leukodystrophies. However, new treatments such as hematopoietic stem cell transplantation have shown promise.

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