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...

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Detalles Bibliográficos
Autores principales: Arranz, Leonor, Aldamiz-Echevarria, Luis
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2015
Materias:
Case Report
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

Internet

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

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