Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human α-L …
JE Wraith, M Beck, R Lane, A Van Der Ploeg… - …, 2007 - publications.aap.org
OBJECTIVE. Our objective was to evaluate the safety, pharmacokinetics, and efficacy of
laronidase in young, severely affected children with mucopolysaccharidosis I. METHODS.
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 scheduled to receive intravenous laronidase at 100 U/kg (0.58
mg/kg) weekly for 52 weeks. Four patients underwent dosage increases to 200 U/kg for the …
laronidase in young, severely affected children with mucopolysaccharidosis I. METHODS.
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 scheduled to receive intravenous laronidase at 100 U/kg (0.58
mg/kg) weekly for 52 weeks. Four patients underwent dosage increases to 200 U/kg for the …
