Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
1
pubmed:dateCreated
2008-12-9
pubmed:abstractText
Gaucher disease is the most common lysosomal storage disease, and the first lysosomal storage disease for which a specific therapy has been developed. Enzyme replacement therapy, with glucocerebrosidase purified from human placentae, was introduced in 1991. Recombinant human glucocerebrosidase, produced by Chinese hamster ovary cells in tissue culture, became available in 1994 and has replaced the placenta-derived product. These therapies have revolutionized the care of patients with type 1 Gaucher disease, reversing many of the pathological consequences of this disease, and preventing further progression. Furthermore, they have served as a model for the treatment of other lysosomal storage diseases and inborn errors of metabolism.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Jan
pubmed:issn
1744-7682
pubmed:author
pubmed:issnType
Electronic
pubmed:volume
9
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
121-31
pubmed:meshHeading
pubmed:year
2009
pubmed:articleTitle
Enzyme replacement therapy for Gaucher disease.
pubmed:affiliation
Children's Memorial Hospital, Division of Genetics, Birth Defects and Metabolism, Department of Pediatrics, Northwestern University, Feinberg School of Medicine, Chicago, Illinois 60614, USA. jcharrow@northwestern.edu
pubmed:publicationType
Journal Article, Review