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Article

[Bone marrow transplantation in mucopolysaccharidosis type I, Hurler-Scheie variety. Metabolic correction and clinical results].

Anales espanoles de pediatria - 1 Oct 1990

Ortega Aramburu J J, Domínguez Luengo C, Olivé Oliveras T, Navarro Fernández C

Abstract excerpt

Bone marrow transplant has proved to be an effective treatment in some hereditary metabolic diseases and, especially, in mucopolysdaccharidosis (MPS). A 9-year-old girl, of consanguineous parents, with MPS Type I, Hurler-Scheie syndrome, received a BMT from her heterozygous, HLA-compatible mother. -L-iduronidase activity in leukocytes and fibroblasts was undetectable and glycosaminoglycans (GAG) excretion in...

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PMID: 2126172

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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Hematopoietic Cell Transplantation for Mucopolysaccharidosis Patients Is Safe and Effective: Results after Implementation of International GuidelinesSemantic proximity Read ↗

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.Semantic proximity Read ↗

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