Impact of enzyme replacement therapy on survival in adults with Pompe disease: Results from a prospective international observational study

Orphanet Journal of Rare Diseases

Volumn 8, Issue 1, 2013, Pages

  Güngör, Deniz ^a   Kruijshaar, Michelle E ^a   Plug, Iris ^a   D'Agostino, Ralph B ^b   Hagemans, Marloes L C ^a   Van Doorn, Pieter A ^a   Reuser, Arnold J J ^a   Van Der Ploeg, Ans T ^a,c  

^a ERASMUS MEDICAL CENTER   (Netherlands)

^b HARVARD CLINICAL RESEARCH INSTITUTE   (United States)

^c SOPHIA CHILDREN S HOSPITAL   (Netherlands)

Author keywords

Acid maltase deficiency; Alglucosidase alfa; Enzyme replacement therapy; Glycogen storage disease type II; Lysosomal storage disease; Pompe disease; Survival

Indexed keywords

ENZYME;

ADULT; AGE; ARTICLE; CLINICAL ARTICLE; CLINICAL EFFECTIVENESS; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DEATH; DISEASE SEVERITY; DRUG EFFICACY; ENZYME REPLACEMENT; FEMALE; FOLLOW UP; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; MALE; OBSERVATIONAL STUDY; PROSPECTIVE STUDY; RESIDENTIAL HOME; SEX DIFFERENCE; SURVIVAL; AGED; GLYCOGEN STORAGE DISEASE TYPE II; MIDDLE AGED; PROCEDURES; VERY ELDERLY; YOUNG ADULT;

ADULT; AGED; AGED, 80 AND OVER; ENZYME REPLACEMENT THERAPY; FEMALE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; MALE; MIDDLE AGED; PROSPECTIVE STUDIES; YOUNG ADULT;

EID: 84875299316     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-8-49     Document Type: Article

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