Early Use of Drastic Therapy

Krabbe's disease, or globoid-cell leukodystrophy, is an inborn error of lipid metabolism first described in 1916 in two children with spasticity who died in infancy and were found to have “diffuse sclerosis” of the brain.1 Prominent features of the brain in affected children were decreased white-matter mass with relatively normal gray matter, generalized demyelination, and clusters of globoid cells in the white matter. Subsequent histopathological analyses have demonstrated that the changes in the white matter are due to the death of the myelin-forming oligodendrocytes in the central nervous system and Schwann cells in the peripheral nervous system, whereas the characteristic . . .