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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
2
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pubmed:dateCreated |
2002-7-22
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pubmed:abstractText |
Gaucher disease is the first lysosomal storage disorder to be treated with macrophage-targeted enzyme replacement therapy. Previous studies in relatively small numbers of patients demonstrated short-term efficacy of this treatment. This study describes the effects of 2 to 5 years of treatment on specific manifestations of type 1 Gaucher disease.
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pubmed:language |
eng
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pubmed:journal |
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pubmed:citationSubset |
AIM
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pubmed:chemical |
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pubmed:status |
MEDLINE
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pubmed:month |
Aug
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pubmed:issn |
0002-9343
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pubmed:author |
pubmed-author:AnderssonHans CHC,
pubmed-author:CharrowJoelJ,
pubmed-author:KaplanPaigeP,
pubmed-author:KolodnyEdwin HEH,
pubmed-author:MistryPramodP,
pubmed-author:PastoresGregoryG,
pubmed-author:RosenbloomBarry EBE,
pubmed-author:ScottC RonaldCR,
pubmed-author:WappnerRebecca SRS,
pubmed-author:WeinrebNeal JNJ,
pubmed-author:ZimranAriA
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pubmed:issnType |
Print
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pubmed:day |
1
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pubmed:volume |
113
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
112-9
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pubmed:dateRevised |
2006-11-15
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pubmed:meshHeading |
pubmed-meshheading:12133749-Adolescent,
pubmed-meshheading:12133749-Adult,
pubmed-meshheading:12133749-Child,
pubmed-meshheading:12133749-Child, Preschool,
pubmed-meshheading:12133749-Cohort Studies,
pubmed-meshheading:12133749-Dose-Response Relationship, Drug,
pubmed-meshheading:12133749-Drug Administration Schedule,
pubmed-meshheading:12133749-Female,
pubmed-meshheading:12133749-Follow-Up Studies,
pubmed-meshheading:12133749-Gaucher Disease,
pubmed-meshheading:12133749-Glucosylceramidase,
pubmed-meshheading:12133749-Hemoglobins,
pubmed-meshheading:12133749-Humans,
pubmed-meshheading:12133749-Infusions, Intravenous,
pubmed-meshheading:12133749-Liver,
pubmed-meshheading:12133749-Male,
pubmed-meshheading:12133749-Pain Measurement,
pubmed-meshheading:12133749-Platelet Count,
pubmed-meshheading:12133749-Registries,
pubmed-meshheading:12133749-Risk Assessment,
pubmed-meshheading:12133749-Spleen,
pubmed-meshheading:12133749-Statistics, Nonparametric,
pubmed-meshheading:12133749-Time Factors,
pubmed-meshheading:12133749-Treatment Outcome
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pubmed:year |
2002
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pubmed:articleTitle |
Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry.
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pubmed:affiliation |
Department of Medicine, University Hospital, Tamarac, Florida 33321, USA. boneal@winning.com
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pubmed:publicationType |
Journal Article,
Research Support, Non-U.S. Gov't
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