Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: An open-label single-center study

Orphanet Journal of Rare Diseases

Volumn 7, Issue 1, 2012, Pages

  De Vries, Juna M ^a   Van Der Beek, Nadine A ^a   Hop, Wim C ^a   Karstens, Francois P ^a   Wokke, John H ^b   De Visser, Marianne ^c   Van Engelen, Baziel G ^d   Kuks, Jan B ^e   Van Der Kooi, Anneke J ^c   Notermans, Nicolette C ^b   Faber, Catharina G ^f   Verschuuren, Jan J ^g   Kruijshaar, Michelle E ^a   Reuser, Arnold J ^a   Van Doorn, Pieter A ^a   Van Der Ploeg, Ans T ^a  

^a ERASMUS MEDICAL CENTER   (Netherlands)

^b UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^c ACADEMIC MEDICAL CENTER   (Netherlands)

^d RADBOUD UNIVERSITY MEDICAL CENTER   (Netherlands)

^e UNIVERSITY MEDICAL CENTER GRONINGEN   (Netherlands)

^f MAASTRICHT UNIVERSITY MEDICAL CENTER   (Netherlands)

^g LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

Acid glucosidase; Alglucosidase alfa; Enzyme replacement therapy; Glycogen storage disease type II; Lung function; Lysosomal storage disorder; Muscle strength; OMIM number 232300; Pompe disease

Indexed keywords

PLACEBO; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE;

ADULT; AGED; ARTICLE; COHORT ANALYSIS; CONTROLLED STUDY; DISEASE COURSE; DRUG EFFICACY; DRUG SAFETY; DYNAMOMETRY; ENZYME REPLACEMENT; FEMALE; FOLLOW UP; FORCED VITAL CAPACITY; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; LUNG FUNCTION; MAJOR CLINICAL STUDY; MALE; MUSCLE FUNCTION; MUSCLE STRENGTH; MUSCLE WEAKNESS; OPEN STUDY; PROSPECTIVE STUDY; RANDOMIZED CONTROLLED TRIAL;

ADULT; AGED; ALPHA-GLUCOSIDASES; COHORT STUDIES; FEMALE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; MALE; MIDDLE AGED; MUSCLE, SKELETAL; PROGNOSIS; RESPIRATION, ARTIFICIAL; RESPIRATORY FUNCTION TESTS;

EID: 84866532475     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-7-73     Document Type: Article

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