Long term survival and cardiopulmonary outcome in children with Hurler syndrome after haematopoietic stem cell transplantation
Sign Up to like & getrecommendations! Published in 2017 at "Journal of Inherited Metabolic Disease"
DOI: 10.1007/s10545-017-0034-6
Abstract: Premature death in untreated children with Hurler syndrome (HS) in the first decade of life is largely due to life-threatening cardiopulmonary complications. We examined the long-term survival and cardiopulmonary outcome in 54 children undergoing haematopoietic… read more here.
Keywords: outcome; term; hurler syndrome; children hurler ... See more keywords
Gene therapy for Hurler syndrome
Sign Up to like & getrecommendations! Published in 2021 at "Journal of Paediatrics and Child Health"
DOI: 10.1111/jpc.15871
Abstract: Several traditional, susceptibleinfectedremoved models have been applied to describe influenza epidemics; however, these models cannot quantitatively assess the efficacy of vaccination. Otani et al.’s study aimed to develop a novel model to virtually reproduce the… read more here.
Keywords: vaccination; transmission; gene therapy; efficacy ... See more keywords
Immune Mediated Cytopenia's Following Cord Blood Transplantation for Hurler's Syndrome: A Failure of Pre-Transplant Immune Suppression
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DOI: 10.1182/blood-2018-99-112995
Abstract: Background: Hurler's syndrome is the most severe phenotype of Mucopolysaccharidosis 1 (MPS1H) with severe deficiency of the lysosomal enzyme alpha-L-iduronidase causing heparan and dermatan sulfate accumulation, CNS and somatic manifestations and premature death. Haematopoeitic Stem… read more here.
Keywords: risk; hurler syndrome; transplant; risk factors ... See more keywords