Abstract
We report successful bone marrow transplantation in a child with a severe form of α-mannosidosis, type I. There was complete resolution of the recurrent sinopulmonary disease and organomegaly, improvement in the bony disease, and stabilization of neurocognitive function.
Original language | English |
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Pages (from-to) | 282-285 |
Number of pages | 4 |
Journal | Journal of Pediatrics |
Volume | 133 |
Issue number | 2 |
DOIs | |
State | Published - 1998 |