Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities. At ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

The Food and Drug Administration on Monday approved a gene therapy for metachromatic leukodystrophy, a devastating genetic disorder that eats away at affected children’s neurons. The medicine, ...

Leukodystrophy refers to a group of rare genetic disorders affecting the central nervous system (CNS). The CNS includes the brain and spinal cord. The outlook for people with these disorders is ...