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Article

Activities of daily living in patients with Hunter syndrome: impact of enzyme replacement therapy and hematopoietic stem cell transplantation.

Molecular genetics and metabolism - 1 Feb 2015

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

Abstract excerpt

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 (ERT) and hematopoietic stem cell transplantation (HSCT). We also explored early signs and symptoms to make early...

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Identifiers

PMID: 25468646
DOI: 10.1016/j.ymgme.2014.11.002
PMCID: PMC4312709

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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