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Enzyme replacement therapy in Hurler syndrome after failure of hematopoietic transplant
The most severe form of Mucopolysaccharosidosis type I (MPS-I), Hurler syndrome, presents with progressive respiratory, cardiac and musculoskeletal symptoms and cognitive deterioration. Treatment includes enzyme replacement therapy (ERT) and/or hematopoietic stem cell transplantation (HSCT). We desc...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Elsevier
2015
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4750618/ https://www.ncbi.nlm.nih.gov/pubmed/26937401 http://dx.doi.org/10.1016/j.ymgmr.2015.04.003 |