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A prospective natural history study of Mucopolysaccharidosis Type IIIA
OBJECTIVES: To characterize the clinical course of mucopolysaccharidosis type IIIA (MPS IIIA), and identified potential endpoints for future treatment trials. STUDY DESIGN: Children with a confirmed diagnosis of MPS IIIA, functioning above a developmental age of 1 year, were followed for up to 2 yea...
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| 出版年: | J Pediatr |
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| 主要な著者: | , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
2016
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4769976/ https://ncbi.nlm.nih.gov/pubmed/26787381 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jpeds.2015.11.079 |
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