Enzyme replacement therapy for Fabry disease: Lessons from two α-galactosidase A orphan products and one FDA approval

Expert Opinion on Biological Therapy

Volumn 4, Issue 7, 2004, Pages 1167-1176

  Desnick, Robert J ^a  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

Agalsidase alfa; Agalsidase beta; Enzyme replacement therapy; Fabry disease; Globotriaosylceramide

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALPHA GALACTOSIDASE; GLOBOTRIAOSYLCERAMIDE; ORPHAN DRUG;

CHILL; CLINICAL TRIAL; DRUG APPROVAL; DRUG EFFICACY; DRUG INDICATION; DRUG MARKETING; DRUG SAFETY; ENZYME REPLACEMENT; FABRY DISEASE; FEVER; FOOD AND DRUG ADMINISTRATION; HUMAN; OUTCOMES RESEARCH; PATHOPHYSIOLOGY; REVIEW; SIDE EFFECT; UNITED STATES;

ADULT; ALPHA-GALACTOSIDASE; ANIMALS; BIOPSY; CELL LINE; CHEMISTRY, PHARMACEUTICAL; CHO CELLS; CLINICAL TRIALS; CRICETINAE; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG APPROVAL; EUROPE; FABRY DISEASE; FEMALE; GENOTYPE; HUMANS; ISOANTIBODIES; ISOENZYMES; KIDNEY; MALE; ORPHAN DRUG PRODUCTION; RESEARCH DESIGN; TREATMENT OUTCOME; TRIHEXOSYLCERAMIDES; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 3142737171     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.4.7.1167     Document Type: Review

References (43)

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