Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human {alpha}-L-Iduronidase (Laronidase)

Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human {alpha}-L-Iduronidase (Laronidase)

Our objective was to evaluate the safety, pharmacokinetics, and efficacy of laronidase in young, severely affected children with mucopolysaccharidosis I. This was a prospective, open-label, multinational study of 20 patients who had mucopolysaccharidosis I and were

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Bibliographische Detailangaben
Veröffentlicht in:Pediatrics (Evanston) 2007-07, Vol.120 (1), p.e37
Hauptverfasser: Wraith, J. Edmond, Beck, Michael, Lane, Roderick, van der Ploeg, Ans, Shapiro, Elsa, Xue, Yong, Kakkis, Emil D, Guffon, Nathalie
Format: Artikel
Sprache:eng
Schlagworte:
Children & youth
Clinical outcomes
Enzymes
Genetic disorders
Hormone replacement therapy
Pediatrics
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Zusammenfassung:Our objective was to evaluate the safety, pharmacokinetics, and efficacy of laronidase in young, severely affected children with mucopolysaccharidosis I. This was a prospective, open-label, multinational study of 20 patients who had mucopolysaccharidosis I and were
ISSN:0031-4005
1098-4275
DOI:10.1542/peds.2006-2156