Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group
Authors:
Open Access
- 1 June 2020
- journal article
- research article
- Published by Elsevier in Molecular Genetics and Metabolism Reports
- Vol. 23, 100578
Abstract
No abstract availableKeywords
Funding Information
- Amicus
- Biomarin
- Sanofi Genzyme
- Shire
- Actelion
- Chiesi Farmaceutici S.p.A.
This publication has 28 references indexed in Scilit:
- Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedureOrphanet Journal of Rare Diseases, 2011
- Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and GreeceAmerican Journal of Medical Genetics Part A, 2010
- Mucopolysaccharidosis type IIIA: Clinical spectrum and genotype‐phenotype correlationsAnnals of Neurology, 2010
- Mucopolysaccharidosis type III (Sanfilippo disease) in Sweden: clinical presentation of 22 children diagnosed during a 30‐year periodActa Paediatrica, 2010
- Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005Bone Marrow Transplantation, 2008
- 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
- Results of the Cord Blood Transplantation Study (COBLT): Outcomes of Unrelated Donor Umbilical Cord Blood Transplantation in Pediatric Patients with Lysosomal and Peroxisomal Storage DiseasesTransplantation and Cellular Therapy, 2006
- Treatment of pediatric patients with Sanfilippo syndrome (MPS IIIA and IIIB) with unrelated umbilical cord blood transplantationTransplantation and Cellular Therapy, 2005
- Bone marrow transplantation for Sanfilippo disease type BJournal of Inherited Metabolic Disease, 1992
- Displacement bone marrow transplantation for some inborn errorsJournal of Inherited Metabolic Disease, 1990