Canavan disease is a rare form of leukodystrophy, a group of conditions that result in the degeneration of white matter in the brain, which is the part of the cerebral cortex filled with myelinated axons. An axon is the part of a nerve cell that conducts neural impulses, allowing neural signals to travel throughout the nervous system. Myelin refers to the fatty coating around axons which helps insulate the axon to make neural impulses travel quickly and efficiently. Loss of myelin, or demyelination, makes travel for neural impulses difficult, ultimately leading to a variety of sensory, motor, and cognitive problems.

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