Hematopoietic Stem-Cell Transplantation in Globoid-Cell Leukodystrophy

Globoid-cell leukodystrophy is an autosomal recessive disease due to greatly diminished or absent activity of the lysosomal enzyme galactocerebrosidase. 1 The disease is characterized by the progressive loss of central and peripheral myelin and by spasticity, dementia, and peripheral neuropathy. It ends in a chronic vegetative state and early death. The more common form begins in early infancy and is rapidly progressive, often leading to death within two years. The late-onset form typically begins later in childhood, has a more insidious onset, and progresses over a period of several years to death. 2 , 3 Hematopoietic stem-cell transplantation has been shown to alter . . .