Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10year combined experience of 2 centres
Author:
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism
Reference18 articles.
1. Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure;de Ru;Orphanet. J. Rare. Dis.,2011
2. Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome;Cox-Brinkman;Bone Marrow Transplant.,2006
3. Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients;Wynn;J. Pediatr.,2009
4. Dimethylmethylene blue-based spectrophotometry of glycosaminoglycans in untreated urine: a rapid screening procedure for mucopolysaccharidoses;de Jong;Clin. Chem.,1989
5. Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning;Boelens;Blood,2013
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