Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report
Open Access
- 7 August 2015
- journal article
- research article
- Published by Springer Science and Business Media LLC in Orphanet Journal of Rare Diseases
- Vol. 10 (1), 1-19
- https://doi.org/10.1186/s13023-015-0313-y
Abstract
No abstract availableKeywords
This publication has 42 references indexed in Scilit:
- Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic LeukodystrophyScience, 2013
- Developing therapeutic approaches for metachromatic leukodystrophyDrug Design, Development and Therapy, 2013
- Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohortBone Marrow Transplantation, 2012
- Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantationProceedings of the National Academy of Sciences of the United States of America, 2012
- Developing treatment options for metachromatic leukodystrophyMolecular Genetics and Metabolism, 2012
- Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: A 5-year follow-up in three affected siblingsMolecular Genetics and Metabolism, 2011
- Clinical and biochemical study of 29 Brazilian patients with metachromatic leukodystrophyJournal of Inherited Metabolic Disease, 2010
- Metachromatic Leukodystrophy: A Case of Triplets With the Late Infantile Variant and a Systematic Review of the LiteratureJournal of Child Neurology, 2009
- Early marrow transplantation in a pre-symptomatic neonate with late infantile metachromatic leukodystrophy does not halt disease progressionBone Marrow Transplantation, 2007
- Nonparametric Estimation from Incomplete ObservationsJournal of the American Statistical Association, 1958