Bobby Gasper, MD, Chief Scientific Officer of Orchard Therapeutics, discusses gene therapy being developed to treat children with metachromatic leukodystrophy (MLD).
MLD is a lysosomal storage disorder characterized by the accumulation of sulfatides in cells. This accumulation largely affects myelin producing cells that leads to the progressive destruction of white matter (leukodystrophy) throughout the nervous system.
Affected individuals show progressive deterioration of cognitive ability, motor and sensory functions. Eventually they lose awareness of their surroundings and become unresponsive.
There is currently no treatment available for this devastating condition.
To learn more about this and other lysosomal storage disorders, click here.