The NCS-LSD cohort study: a description of the methods and analyses used to assess the long-term effectiveness of enzyme replacement therapy and substrate reduction therapy in patients with lysosomal storage disorders

Journal of Inherited Metabolic Disease

Volumn 37, Issue 6, 2014, Pages 939-944

  Henley, W E ^a   Anderson, L J ^a   Wyatt, K M ^a   Nikolaou, V ^a   Anderson, R ^a   Logan, S ^a  

^a UNIVERSITY OF EXETER MEDICAL SCHOOL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ADOLESCENT; ADULT; ARTICLE; CHILD; DISEASE COURSE; ENZYME REPLACEMENT; FABRY DISEASE; GAUCHER DISEASE; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; LONG TERM CARE; LYSOSOME STORAGE DISEASE; MAJOR CLINICAL STUDY; NIEMANN PICK DISEASE; OUTCOME ASSESSMENT; CLINICAL TRIAL; ENGLAND; FEMALE; LONGITUDINAL STUDY; LYSOSOMAL STORAGE DISEASES; MALE; MULTICENTER STUDY; PROCEDURES; PROSPECTIVE STUDY; REGRESSION ANALYSIS; RETROSPECTIVE STUDY; TREATMENT OUTCOME;

ADULT; CHILD; ENGLAND; ENZYME REPLACEMENT THERAPY; FEMALE; HUMANS; LONGITUDINAL STUDIES; LYSOSOMAL STORAGE DISEASES; MALE; PROSPECTIVE STUDIES; REGRESSION ANALYSIS; RETROSPECTIVE STUDIES; TREATMENT OUTCOME;

EID: 84912026258     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-014-9679-6     Document Type: Article

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