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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Bibliografiska uppgifter
I publikationen:Mol Genet Metab
Huvudupphovsmän: 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
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2014
Ämnen:
Article
Länkar: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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