Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation.
Lancet
; 2(8249): 709-12, 1981 Oct 03.
Article
en En
| MEDLINE
| ID: mdl-6116856
ABSTRACT
A one-year-old boy with type I H mucopolysaccharidosis (Hurler's disease) was given a bone-marrow transplant (BMT) from his mother in an attempt to replace the deficient enzyme, alpha-L-iduronidase (iduronidase). These is definite evidence of engraftment, the enzyme activity of the recipient's leucocytes reaching heterozygote levels within 37 days of the BMT. Graft-versus-host disease (GVHD) developed but was partially controlled by steroids. From 3-4 months after graft until the present (13 months after the graft) iduronidase activity has been present in the serum and the urine and there has been evidence of considerable degradation of glycosaminoglycans excreted in the urine. The hepatosplenomegaly has disappeared, corneal clouding has cleared, and deterioration in the child's development seems to have been arrested.
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Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Trasplante de Médula Ósea
/
Mucopolisacaridosis I
Límite:
Humans
/
Infant
/
Male
Idioma:
En
Revista:
Lancet
Año:
1981
Tipo del documento:
Article