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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
3
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pubmed:dateCreated |
2006-9-4
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pubmed:abstractText |
Fabry disease is an X-linked multisystem disorder. Enzyme-replacement therapy in adults has limited efficacy in treating major sequelae of advanced Fabry disease, such as kidney failure or stroke. This prompted a study of the safety and efficacy of enzyme replacement at an earlier stage of Fabry 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 |
Sep
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pubmed:issn |
1098-4275
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pubmed:author |
pubmed-author:BeckMichaelM,
pubmed-author:BradyRoscoe ORO,
pubmed-author:ClarkeJoe T RJT,
pubmed-author:KampmannChristophC,
pubmed-author:LienY HowardYH,
pubmed-author:PastoresGregoryG,
pubmed-author:RiesMarkusM,
pubmed-author:RobinsonChevaliaC,
pubmed-author:SchiffmannRaphaelR,
pubmed-author:SchlaggarBradley LBL,
pubmed-author:TimmonsMargaretM,
pubmed-author:WhybraCatharinaC
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pubmed:issnType |
Electronic
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pubmed:volume |
118
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
924-32
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pubmed:dateRevised |
2006-11-15
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pubmed:meshHeading |
pubmed-meshheading:16950982-Adolescent,
pubmed-meshheading:16950982-Antibody Formation,
pubmed-meshheading:16950982-Autonomic Nervous System,
pubmed-meshheading:16950982-Child,
pubmed-meshheading:16950982-Fabry Disease,
pubmed-meshheading:16950982-Female,
pubmed-meshheading:16950982-Heart Rate,
pubmed-meshheading:16950982-Humans,
pubmed-meshheading:16950982-Isoenzymes,
pubmed-meshheading:16950982-Male,
pubmed-meshheading:16950982-Treatment Outcome,
pubmed-meshheading:16950982-alpha-Galactosidase
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pubmed:year |
2006
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pubmed:articleTitle |
Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease.
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pubmed:affiliation |
Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892-1260, USA.
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pubmed:publicationType |
Journal Article,
Clinical Trial,
Research Support, N.I.H., Intramural
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