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Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report

BACKGROUND: Metachromatic Leukodystrophy (MLD) is a rare, fatal demyelinating disorder with limited treatment options. Published outcomes after hematopoietic stem cell transplantation (HSCT) are scant and mixed. We report survival and function following HSCT for a large, single-center MLD cohort. ME...

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Detalles Bibliográficos
Autores principales: Boucher, Alexander A., Miller, Weston, Shanley, Ryan, Ziegler, Richard, Lund, Troy, Raymond, Gerald, Orchard, Paul J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4545855/
https://www.ncbi.nlm.nih.gov/pubmed/26245762
http://dx.doi.org/10.1186/s13023-015-0313-y