Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis

Journal of Neurology

Volumn 264, Issue 4, 2017, Pages 621-630

  Schoser, Benedikt ^a   Stewart, Andrew ^b   Kanters, Steve ^c,d   Hamed, Alaa ^b   Jansen, Jeroen ^d   Chan, Keith ^d   Karamouzian, Mohammad ^c,d   Toscano, Antonio ^e  

^a LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

^b GENZYME CORPORATION   (United States)

^c UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^d Redwood Outcomes   (Canada)

^e UNIVERSITY OF MESSINA   (Italy)

Author keywords

Alglucosidase alfa; Enzyme replacement therapy; Glycogen storage disease type 2; Late onset Pompe disease (LOPD); Systematic review

Indexed keywords

RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; ALPHA GLUCOSIDASE; GAA PROTEIN, HUMAN;

ACCOUNTING; COCHRANE LIBRARY; DATA EXTRACTION; DRUG EFFICACY; EMBASE; ENZYME REPLACEMENT; FOLLOW UP; FORCED VITAL CAPACITY; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; LATE ONSET POMPE DISEASE; LONG TERM CARE; MEDLINE; META ANALYSIS; MOBILIZATION; MORTALITY RATE; MOTOR PERFORMANCE; MULTICENTER STUDY (TOPIC); OUTCOME ASSESSMENT; POISSON DISTRIBUTION; PRIORITY JOURNAL; QUALITY OF LIFE; RESPIRATORY FUNCTION; REVIEW; SIX MINUTE WALK TEST; SURVIVAL RATE; SYSTEMATIC REVIEW; TREATMENT DURATION; TREATMENT OUTCOME; GLYCOGEN STORAGE DISEASE TYPE II; MORTALITY;

ALPHA-GLUCOSIDASES; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; TREATMENT OUTCOME;

EID: 84976507595     PISSN: 03405354     EISSN: 14321459     Source Type: Journal    
DOI: 10.1007/s00415-016-8219-8     Document Type: Review

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