TY - JOUR
T1 - Mixed donor chimerism and low level iduronidase expression may be adequate for neurodevelopmental protection in Hurler syndrome
AU - Conway, Jennifer
AU - Dyack, Sarah
AU - Crooks, Bruce N.A.
AU - Fernandez, Conrad V.
PY - 2005/7
Y1 - 2005/7
N2 - Hurler syndrome is a lysosomal storage disease resulting in fatal cardiac or neurologic sequelae unless alpha-iduronidase production is reconstituted with hematopoietic stem cell transplantation. We report on a 4-year, 6-month-old boy with mixed donor chimerism and low enzyme levels but a normal neurodevelopmental trajectory.
AB - Hurler syndrome is a lysosomal storage disease resulting in fatal cardiac or neurologic sequelae unless alpha-iduronidase production is reconstituted with hematopoietic stem cell transplantation. We report on a 4-year, 6-month-old boy with mixed donor chimerism and low enzyme levels but a normal neurodevelopmental trajectory.
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U2 - 10.1016/j.jpeds.2005.03.005
DO - 10.1016/j.jpeds.2005.03.005
M3 - Article
C2 - 16027706
AN - SCOPUS:22144442824
SN - 0022-3476
VL - 147
SP - 106
EP - 108
JO - Journal of Pediatrics
JF - Journal of Pediatrics
IS - 1
ER -