Activities of Daily Living in patients with Hunter syndrome: Impact of enzyme replacement therapy and hematopoietic stem cell transplantation

The aim of this study was to assess the Activities of Daily Living (ADL) in patients with Hunter syndrome (mucopolysaccharidosis II; MPS II) using a newly designed ADL questionnaire. We applied the questionnaire to evaluate clinical phenotypes and therapeutic efficacies of enzyme replacement therapy...

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Publicado en:Mol Genet Metab
Autores principales: Tanjuakio, Julian, Suzuki, Yasuyuki, Patel, Pravin, Yasuda, Eriko, Kubaski, Francyne, Tanaka, Akemi, Yabe, Hiromasa, Mason, Robert W., Montaño, Adriana M., Orii, Kenji E., Orii, Koji O., Fukao, Toshiyuki, Orii, Tadao, Tomatsu, Shunji
Formato: Artigo
Lenguaje:Inglês
Publicado: 2014
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4312709/
https://ncbi.nlm.nih.gov/pubmed/25468646
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2014.11.002
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