Canavan Disease - Pipeline Review, H1 2017

Canavan disease is a gene-linked neurological disorder in which the brain degenerates into spongy tissue riddled with microscopic fluid-filled spaces. Cause includes a mutation in the gene which directs the production of the enzyme aspartoacylase (ASPA) allows the buildup of N-acetylaspartic acid (NAA) in the brain. The buildup of NAA causes damage to myelin.