Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future
Open Access
- 14 February 2019
- journal article
- review article
- Published by Elsevier BV in Transplantation and Cellular Therapy
- Vol. 25 (7), e226-e246
- https://doi.org/10.1016/j.bbmt.2019.02.012
Abstract
No abstract availableFunding Information
- Japan Agency for Medical Research and Development (JP18gk0110017)
- National Institute of General Medical Sciences (GM114736)
This publication has 182 references indexed in Scilit:
- Prevalence and Development of Orthopaedic Symptoms in the Dutch Hurler Patient Population after Haematopoietic Stem Cell TransplantationPublished by Springer Science and Business Media LLC ,2012
- The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensusEuropean Journal of Pediatrics, 2011
- Combined Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation in Mucopolysacharidosis Type VIPublished by Springer Science and Business Media LLC ,2011
- Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VIMolecular Genetics and Metabolism, 2011
- Mucopolysaccharidosis VIOrphanet Journal of Rare Diseases, 2010
- Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomesBlood, 2008
- The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UKOrphanet Journal of Rare Diseases, 2008
- Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis IJCI Insight, 2008
- Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapyEuropean Journal of Pediatrics, 2007
- NanomedicineClinical Pharmacokinetics, 2006