Clinical features and predictors for disease natural progression in adults with Pompe disease: A nationwide prospective observational study

Orphanet Journal of Rare Diseases

Volumn 7, Issue 1, 2012, Pages

  Van Der Beek, Nadine A M E ^a,b   De Vries, Juna M ^a,b   Hagemans, Marloes L C ^b   Hop, Wim C J ^a   Kroos, Marian A ^a   Wokke, John H J ^c   De Visser, Marianne ^d   Van Engelen, Baziel G M ^e   Kuks, Jan B M ^f   Van Der Kooi, Anneke J ^d   Notermans, Nicolette C ^c   Faber, Karin G ^g   Verschuuren, Jan J G M ^h   Reuser, Arnold J J ^a   Van Der Ploeg, Ans T ^b   Van Doorn, Pieter A ^a  

^a ERASMUS MEDICAL CENTER   (Netherlands)

^b SOPHIA CHILDREN S HOSPITAL   (Netherlands)

^c UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^d ACADEMIC MEDICAL CENTER   (Netherlands)

^e RADBOUD UNIVERSITY MEDICAL CENTER   (Netherlands)

^f UNIVERSITY MEDICAL CENTER GRONINGEN   (Netherlands)

^g MAASTRICHT UNIVERSITY MEDICAL CENTER   (Netherlands)

^h LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

Acid glucosidase; Disease progression; Glycogen storage disease type II; Lysosomal storage disorder; Natural course; OMIM number 232300; Prognostic factors

Indexed keywords

ADULT; AGED; ARTICLE; BULBAR PARALYSIS; CLINICAL FEATURE; DISEASE DURATION; DYNAMOMETRY; FEMALE; FOLLOW UP; FORCED VITAL CAPACITY; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; LUNG FUNCTION TEST; MAJOR CLINICAL STUDY; MALE; MOTOR PERFORMANCE; MUSCLE STRENGTH; MUSCLE WEAKNESS; OBSERVATIONAL STUDY; PREDICTION; PROSPECTIVE STUDY; SCAPULAR WINGING;

ADULT; AGED; ALPHA-GLUCOSIDASES; DISEASE PROGRESSION; FEMALE; GLYCOGEN STORAGE DISEASE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; MALE; MIDDLE AGED; PROSPECTIVE STUDIES;

EID: 84868644570     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-7-88     Document Type: Article

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