
07/10/2025
Canavan disease (CD) is a fatal genetic leukodystrophy caused by mutations in the ASPA gene, leading to accumulation of N-acetylaspartate (NAA) and breakdown of myelin, the insulating sheath around nerve fibers. Affected children suffer progressive neurological impairment, blindness, seizures, and early death.
MYR-101 gene therapy reduces N-acetylaspartate, increases myelin, and improves development in Canavan disease patients.