Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study

Journal of Inherited Metabolic Disease

Volumn 37, Issue 6, 2014, Pages 945-952

  Anderson, L J ^a   Henley, W ^a   Wyatt, K M ^a   Nikolaou, V ^a   Waldek, S ^c   Hughes, D A ^d   Lachmann, R H ^b   Logan, S ^a  

^a UNIVERSITY OF EXETER MEDICAL SCHOOL   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON HOSPITALS   (United Kingdom)

^c SALFORD ROYAL NHS FOUNDATION TRUST   (United Kingdom)

^d UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE;

ADOLESCENT; ADULT; AGED; ANAPHYLAXIS; ARTICLE; BODY MASS; COHORT ANALYSIS; DRUG EFFICACY; ENZYME REPLACEMENT; FEMALE; FORCED VITAL CAPACITY; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; LUNG VENTILATION; MAJOR CLINICAL STUDY; MALE; MIDDLE AGED; MUSCLE STRENGTH; PATIENT MOBILITY; RESPIRATORY FUNCTION; TREATMENT DURATION; UNITED KINGDOM; YOUNG ADULT; CLINICAL TRIAL; ENGLAND; GLYCOGEN STORAGE DISEASE TYPE II; LONGITUDINAL STUDY; MULTICENTER STUDY; ONSET AGE; PROCEDURES; PROSPECTIVE STUDY; RETROSPECTIVE STUDY; TREATMENT OUTCOME; WALKING;

ADOLESCENT; ADULT; AGE OF ONSET; AGED; BODY MASS INDEX; ENGLAND; ENZYME REPLACEMENT THERAPY; FEMALE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; LONGITUDINAL STUDIES; MALE; MIDDLE AGED; MUSCLE STRENGTH; PROSPECTIVE STUDIES; RETROSPECTIVE STUDIES; TREATMENT OUTCOME; WALKING; YOUNG ADULT;

EID: 84912014167     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-014-9728-1     Document Type: Article

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