Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres

Haematopoietic stem cell transplantation is the treatment of choice for the severe form of Mucopolysaccharidosis Type I, or Hurler syndrome. In many centres standard practice is to deliver enzyme replacement therapy alongside haematopoietic stem cell transplantation to improve the condition of the p...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab
Main Authors: Ghosh, Arunabha, Miller, Weston, Orchard, Paul J., Jones, Simon A., Mercer, Jean, Church, Heather J., Tylee, Karen, Lund, Troy, Bigger, Brian W., Tolar, Jakub, Wynn, Robert F.
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5177791/
https://ncbi.nlm.nih.gov/pubmed/26832957
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2016.01.011
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