Enzyme replacement therapy: Lessons learned and emerging questions

Expert Opinion on Orphan Drugs

Volumn 3, Issue 3, 2015, Pages 293-305

  Jurecka, Agnieszka ^a,b   Tylki Szymańska, Anna ^a  

^a CHILDREN'S MEMORIAL HEALTH INSTITUTE   (Poland)

^b UNIVERSITY OF GDA SK   (Poland)

Author keywords

Enzyme replacement therapy; Fabry disease; Gaucher disease; Mucopolysaccharidosis; Pompe disease

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALGLUCERASE; AUTOANTIBODY; ELOSULFASE ALFA; GALSULFASE; IDURONATE 2 SULFATASE; IMIGLUCERASE; LARONIDASE; LYSOSOME ENZYME; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; SOMATOMEDIN B RECEPTOR;

ANTIBODY PRODUCTION; ARTHROPATHY; BLOOD BRAIN BARRIER; CENTRAL NERVOUS SYSTEM; COST EFFECTIVENESS ANALYSIS; DISEASE COURSE; DRUG EFFICACY; ENZYME REPLACEMENT; FABRY DISEASE; GAUCHER DISEASE; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HURLER SYNDROME; LYSOSOME STORAGE DISEASE; MORQUIO SYNDROME; MUCOPOLYSACCHARIDOSIS; NEWBORN SCREENING; PATHOPHYSIOLOGY; PRIORITY JOURNAL; REVIEW; SYMPTOMATOLOGY; TREATMENT DURATION; TREATMENT OUTCOME; TREATMENT RESPONSE; VALVULAR HEART DISEASE;

EID: 84923570367     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2015.1017469     Document Type: Review

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