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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...

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Detalles Bibliográficos
Autores principales: Tomita, Kazuyoshi, Okamoto, Shungo, Seto, Toshiyuki, Hamazaki, Takashi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
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