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Real world long-term outcomes in patients with mucopolysaccharidosis type II: A retrospective cohort study
We investigated the decline of activities of daily living with symptomatic progression in patients with mucopolysaccharidosis type II (MPS II) and investigated the associated factors. Clinical data were retrospectively collected from the medical records of 28 patients with MPS II who visited our hos...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8554623/ https://www.ncbi.nlm.nih.gov/pubmed/34745890 http://dx.doi.org/10.1016/j.ymgmr.2021.100816 |