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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Bibliografski detalji
Izdano u:Mol Genet Metab
Glavni autori: 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
Format: Artigo
Jezik:Inglês
Izdano: 2014
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Article
Online pristup: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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