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

Molecular Genetics and Metabolism

Volumn 114, Issue 2, 2015, Pages 161-169

  Tanjuakio, Julian ^a   Suzuki, Yasuyuki ^b   Patel, Pravin ^a   Yasuda, Eriko ^a   Kubaski, Francyne ^a,c   Tanaka, Akemi ^d   Yabe, Hiromasa ^e   Mason, Robert W ^a   Montaño, Adriana M ^f,g   Orii, Kenji E ^h   Orii, Koji O ⁱ   Fukao, Toshiyuki ⁱ   Orii, Tadao ⁱ   Tomatsu, Shunji ^a,i  

^a NEMOURS ALFRED I DUPONT HOSPITAL FOR CHILDREN   (United States)

^b GIFU UNIVERSITY   (Japan)

^c UNIVERSITY OF DELAWARE   (United States)

^d OSAKA CITY UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^e TOKAI UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^f SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^g Department of Biochemistry and Molecular Biology ^*  (United States)

^h GIFU UNIVERSITY HOSPITAL   (Japan)

ⁱ GIFU UNIVERSITY SCHOOL OF MEDICINE   (Japan)

Author keywords

Activities of daily living; Clinical phenotype; Enzyme replacement therapy; Hematopoietic stem cell transplantation; Hunter syndrome

Indexed keywords

ACTIVITY OF DAILY LIVING ASSESSMENT; ADL DISABILITY; ADOLESCENT; ADULT; ARTICLE; CHILD; CLINICAL ASSESSMENT; CLINICAL EVALUATION; COGNITION; COMPARATIVE STUDY; CONTROLLED STUDY; DAILY LIFE ACTIVITY; DISEASE SEVERITY; EARLY DIAGNOSIS; ENZYME REPLACEMENT; FEASIBILITY STUDY; FEMALE; FUNCTIONAL INDEPENDENCE MEASURE; GROUPS BY AGE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; MAJOR CLINICAL STUDY; MALE; MOVEMENT (PHYSIOLOGY); PHENOTYPIC VARIATION; PHYSICAL DISEASE BY BODY FUNCTION; PRESCHOOL CHILD; PRIORITY JOURNAL; QUESTIONNAIRE; SCORING SYSTEM; INFANT; JAPAN; MIDDLE AGED; MUCOPOLYSACCHARIDOSIS II; PHENOTYPE; STANDARDS; YOUNG ADULT;

LEVO IDURONIDASE;

ACTIVITIES OF DAILY LIVING; ADOLESCENT; ADULT; CHILD; CHILD, PRESCHOOL; COGNITION; ENZYME REPLACEMENT THERAPY; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IDURONIDASE; INFANT; JAPAN; MALE; MIDDLE AGED; MUCOPOLYSACCHARIDOSIS II; PHENOTYPE; SURVEYS AND QUESTIONNAIRES; YOUNG ADULT;

EID: 84921644517     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2014.11.002     Document Type: Article

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