Article
Laronidase for cardiopulmonary disease in Hurler syndrome 12 years after bone marrow transplantation.
Pediatrics - 1 Nov 2010
Valayannopoulos Vassili, de Blic Jacques, Mahlaoui Nizar, Stos Bertrand, Jaubert Francis, Bonnet Damien, Fischer Alain, de Lonlay Pascale
Abstract excerpt
A patient with severe mucopolysaccharidosis type I (Hurler syndrome) underwent bone marrow transplantation twice (at the ages of 2 and 2.5 years), both times with his HLA-identical heterozygous brother as the donor. Between the ages of 10 and 14 years, despite 92% donor engraftment and 50% normal α-L-iduronidase activity, he developed progressive respiratory failure with severe pulmonary arterial hypertension,...
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