Long term disease burden post-transplantation: three decades of observations in 25 Hurler patients successfully treated with hematopoietic stem cell transplantation (HSCT)

BACKGROUND: Mucopolysaccharidosis type I-Hurler syndrome (MPSI-H) is a lysosomal storage disease characterized by severe physical symptoms and cognitive decline. Early treatment with hematopoietic cell transplant (HSCT) is critical to the survival of these patients. While survival rates and short-te...

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Detalles Bibliográficos
Autores principales: Guffon, N., Pettazzoni, M., Pangaud, N., Garin, C., Lina-Granade, G., Plault, C., Mottolese, C., Froissart, R., Fouilhoux, A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7847591/
https://www.ncbi.nlm.nih.gov/pubmed/33517895
http://dx.doi.org/10.1186/s13023-020-01644-w

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