Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: Clinical, biochemical, and pathological improvements

Mucopolysaccharidosis type I (MPS I; Hurler syndrome) is a lysosomal storage disease caused by a deficiency of the enzyme α-l-iduronidase which affects multiple organs such as central nervous system (CNS), skeletal system, and physical appearance. Hematopoietic stem cell transplantation (HSCT) is re...

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Detalles Bibliográficos
Autores principales: Yasuda, Eriko, Mackenzie, William G., Ruhnke, Kristen D., Shimada, Tsutomu, Mason, Robert W., Zustin, Jozef, Martin, Paul L., Thacker, Mihir M., Orii, Tadao, Sai, Yoshimichi, Tomatsu, Shunji
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2015
Materias:
SI:Therapy
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4335359/
https://www.ncbi.nlm.nih.gov/pubmed/25709894
http://dx.doi.org/10.1016/j.ymgmr.2014.12.006

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4335359/
https://www.ncbi.nlm.nih.gov/pubmed/25709894
http://dx.doi.org/10.1016/j.ymgmr.2014.12.006

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