Five-Year Follow-Up of Two Siblings with Aspartylglucosaminuria Undergoing Allogeneic Stem-Cell Transplantation from Unrelated Donors

Abstract

The importance of long-term follow-up of children after ASCT in this rare, very slowly progressive lysosomal disease must be emphasized. We report that none of the children had lost any capabilities since the transplantation; moreover, an improvement is shown in biochemical markers and MRI white-matter signals, suggesting a beneficial effect.