相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。Initial report from the Hunter Outcome Survey
J. Edmond Wraith et al.
GENETICS IN MEDICINE (2008)
Safety and efficacy of enzyme replacement therapy with agalsidase beta: An international, open-label study in pediatric patients with Fabry disease
J. Edmond Wraith et al.
JOURNAL OF PEDIATRICS (2008)
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI:: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase
Paul Harmatz et al.
MOLECULAR GENETICS AND METABOLISM (2008)
Recognition and diagnosis of Mucopolysaccharidosis II (Hunter syndrome)
Rick Martin et al.
PEDIATRICS (2008)
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
J. Edmond Wraith et al.
EUROPEAN JOURNAL OF PEDIATRICS (2008)
Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease
Markus Ries et al.
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A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)
Joseph Muenzer et al.
GENETICS IN MEDICINE (2006)
Enzyme replacement therapy for mucopolysaccharidosis VI: A Phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study
Paul Harmatz et al.
JOURNAL OF PEDIATRICS (2006)
Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome):: Results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase
P Harmatz et al.
PEDIATRICS (2005)
Enzyme-replacement therapy in mucopolysaccharidosis I.
ED Kakkis et al.
NEW ENGLAND JOURNAL OF MEDICINE (2001)