Fabry disease in children and response to enzyme replacement therapy: Results from the Fabry Outcome Survey

Clinical Genetics

Volumn 81, Issue 5, 2012, Pages 485-490

  Ramaswami, U ^a   Parini, R ^b   Pintos Morell, G ^c   Kalkum, G ^d   Kampmann, C ^d   Beck, M ^d  

^a CAMBRIDGE UNIVERSITY HOSPITALS NHS FOUNDATION TRUST   (United Kingdom)

^b SAN GERARDO HOSPITAL   (Italy)

^c Servei d'Hematologia   (Spain)

^d JOHANNES GUTENBERG UNIVERSITY   (Germany)

Author keywords

Agalsidase alfa; Children; Enzyme replacement therapy; Fabry disease

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA;

ABDOMINAL PAIN; ADOLESCENT; ARTICLE; CHILD; CHILDHOOD DISEASE; CONSTIPATION; DIARRHEA; DRUG EFFICACY; DRUG HYPERSENSITIVITY; DRUG SAFETY; DRUG SUBSTITUTION; DRUG WITHDRAWAL; ENZYME REPLACEMENT; FABRY DISEASE; FEMALE; FOLLOW UP; GASTROINTESTINAL DISEASE; HEART LEFT VENTRICLE VOLUME; HUMAN; KIDNEY FUNCTION; MAJOR CLINICAL STUDY; MALE; PAIN; PRESCHOOL CHILD; PRIORITY JOURNAL; SCHOOL CHILD; SYMPTOM; TINNITUS; TREATMENT DURATION; TREATMENT RESPONSE; UNSPECIFIED SIDE EFFECT; VOMITING;

ADOLESCENT; ALPHA-GALACTOSIDASE; CHILD; CHILD, PRESCHOOL; ENZYME REPLACEMENT THERAPY; FABRY DISEASE; FEMALE; HUMANS; MALE; TIME FACTORS; TREATMENT OUTCOME;

EID: 84859616858     PISSN: 00099163     EISSN: 13990004     Source Type: Journal    
DOI: 10.1111/j.1399-0004.2011.01671.x     Document Type: Article

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