Zebra of the Week: Metachromatic leukodystrophy

Metachromatic leukodystrophy (MLD) is a debilitating, rare genetic disease that affects the brain and nervous system. It's caused by a deficiency of an enzyme called arylsulfatase A (ARSA), leading to a buildup of sulfatides in the cells. This buildup causes damage to the central and peripheral nervous system, manifesting with loss of motor and cognitive function and early death. This week the FDA approved atidarsagene autotemcel (Lenmeldy), the first gene therapy indicated for the treatment of children with early-onset MLD.