Ten years of the international Pompe survey: patient reported outcomes as a reliable tool for studying treated and untreated children and adults with non-classic Pompe disease

Journal of Inherited Metabolic Disease

Volumn 38, Issue 3, 2015, Pages 495-503

  van der Meijden, J C ^a   Gungor D ^a   Kruijshaar, M E ^a   Muir, A D J ^b   Broekgaarden, H A ^c   van der Ploeg, A T ^a  

^a ERASMUS MEDICAL CENTER   (Netherlands)

^b Association for Glycogen Storage Disease   (United Kingdom)

^c Vereniging Spierziekten Nederland   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA GLUCOSIDASE;

ADOLESCENT; ADULT; AGED; CHILD; COHORT ANALYSIS; ENZYME REPLACEMENT; FATIGUE; FEMALE; GLYCOGEN STORAGE DISEASE TYPE II; HUMAN; INFANT; MALE; MIDDLE AGED; NEWBORN; OUTCOME ASSESSMENT; PATIENT PARTICIPATION; PRESCHOOL CHILD; PROCEDURES; QUALITY OF LIFE; SELF REPORT; VERY ELDERLY; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; AGED, 80 AND OVER; ALPHA-GLUCOSIDASES; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; ENZYME REPLACEMENT THERAPY; FATIGUE; FEMALE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; INFANT; INFANT, NEWBORN; MALE; MIDDLE AGED; PATIENT OUTCOME ASSESSMENT; PATIENT PARTICIPATION; QUALITY OF LIFE; SELF REPORT; YOUNG ADULT;

EID: 84939872313     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-014-9751-2     Document Type: Review

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