Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years...
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)
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Evanston: Am Acad Pediatrics
Journal title
Language
English
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Publisher
Evanston: Am Acad Pediatrics
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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 <5 years old (16 with Hurler syndrome, 4 with Hurler-Scheie syndrome) and were sched...
Alternative Titles
Full title
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)
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TN_cdi_proquest_journals_228417193
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_228417193
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ISSN
0031-4005
E-ISSN
1098-4275
DOI
10.1542/peds.2006-2156
