The Leukodystrophies are a group of rare genetic disorders that affect the central nervous system by disrupting the growth or maintenance of the myelin sheath that insulates nerve cells.

Myelin, sometimes referred to as “white matter” because of its white, fatty appearance, protects and insulates nerve cells responsible for the rapid transmission of electrical signals between neurons. When that transmission is disrupted, it leads to a central nervous system that does not function properly.

In fact, the word Leukodystrophy comes from the Greek words Leuko (meaning white), trophy (meaning growth), and dys (meaning ill). Together, these words describe a set of diseases that affect the growth or maintenance of the white matter (myelin).

Leukodystrophy is a family of diseases that are progressive, have no known treatment, and have no known cure. They are known as “orphan diseases” because they are so rare, they have few financial resources supporting research and, as a result, little scientific activity behind that research.

Leukodystrophies are mostly inherited disorders, meaning that it is passed on from parent to child. The Leukodystrophies share some common features with multiple sclerosis (MS). However, the cause is different; whereas Leukodystrophies are generally caused by a defect in one of the genes involved with the growth or maintenance of the myelin, MS is thought to be caused by an attack on the myelin by the body’s own immune system.

Extensively more information can be found through the United Leukodystrophy Foundation and the Leukodystrophy Care Network.

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