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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Mol Genet Metab
Egile Nagusiak: 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
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 2014
Gaiak:
Article
Sarrera elektronikoa: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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