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...

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書目詳細資料
發表在:Mol Genet Metab Rep
Main Authors: Eisengart, Julie B., Jarnes, Jeanine, Ahmed, Alia, Nestrasil, Igor, Ziegler, Richard, Delaney, Kathleen, Shapiro, Elsa, Whitley, Chester
格式: Artigo
語言:Inglês
出版: Elsevier 2017
主題:
Case Report
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC5622996/
https://ncbi.nlm.nih.gov/pubmed/28983455
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2017.07.012
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