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Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase)

The Journal of Pediatrics · 2004 · Vol. 144(5) · pp. 581–588
J. E. WraithL. ClarkeMichael BeckEdwin H. KolodnyGregory M. PastoresJoseph MuenzerDavid M. RapoportKenneth I. BergerStuart J. SwiedlerEmil KakkisTanja BraakmanElenie ChadbourneKaren Walton–BowenGerald F. Cox
Lysosomal Storage Disorders ResearchCystic Fibrosis Research AdvancesTrypanosoma species research and implicationsMedicineMucopolysaccharidosis IEnzyme replacement therapyPlaceboInternal medicineMucopolysaccharidosisGastroenterologyMucopolysaccharidosis type IAnesthesiaPathology

MeSH terms

AdolescentAdultAnalysis of VarianceChildDouble-Blind MethodFemaleHumansIduronidaseMucopolysaccharidosis IMaleRecombinant ProteinsRespiratory InsufficiencyStatistics, Nonparametric
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542
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20.10
field-weighted impact
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36
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100%
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References
The Metabolic and Molecular Bases of Inherited Disease
Medical Entomology and Zoology · 1995 · 12,175 citations
Spirometric Reference Values from a Sample of the General U.S. Population
American Journal of Respiratory and Critical Care Medicine · 1999 · 4,237 citations
Reference Equations for the Six-Minute Walk in Healthy Adults
American Journal of Respiratory and Critical Care Medicine · 1998 · 2,171 citations
Standardization of Spirometry, 1994 Update. American Thoracic Society
American Journal of Respiratory and Critical Care Medicine · 1995 · 6,590 citations
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Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase) · Scinovex