Definition

Leukodystrophy refers to a group of rare genetic disorders affecting the central and peripheral nervous systems. They are neurodegenerative diseases characterized by abnormalities in myelin, the fatty substance that surrounds, insulates, and facilitates the function of nerve cells.

Description

Leukodystrophy derives from two Greek words; "leuko" means white, referring to the white matter (myelin) of the nervous system, and "dystrophy" means abnormal growth or development. Myelin insulates, or sheaths, nerve cells, helping them to transmit electrical nerve signals. It is a complex substance composed of a number of fat and protein molecules. Without myelin, nerve cells cease to function and eventually die. It also covers the spinal cord and the long nerve cell projections, known as axons, which innervate all of the peripheral tissues.

More than 15 different types of leukodystrophy have been...