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Long-term cognitive and somatic outcomes of enzyme replacement therapy in untransplanted Hurler syndrome
Mucopolysaccharidosis type I (MPS I) was added to the Recommended Uniform Screening Panel for newborn screening in 2016, highlighting recognition that early treatment of MPS I is critical to stem progressive, irreversible disease manifestations. Enzyme replacement therapy (ERT) is an approved treatm...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5622996/ https://www.ncbi.nlm.nih.gov/pubmed/28983455 http://dx.doi.org/10.1016/j.ymgmr.2017.07.012 |