Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses-Reference-Cited by-同舟云学术

Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses

Published:2020-08 Issue:4 Volume:130 Page:255-261
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Jones Simon A.,Marsden Deborah,Koutsoukos Tony,Sniadecki Jennifer,Tylee Karen,Phillippo Sarah,Kakkis Emil

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference36 articles.

1. Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I;Kakkis;Biochem. Mol. Med.,1996

2. The mucopolysaccharidoses;Neufeld,2001

3. Enzyme-replacement therapy in mucopolysaccharidosis I;Kakkis;N. Engl. J. Med.,2001

4. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase);Wraith;J. Pediatr.,2004

5. 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);Wraith;Pediatrics,2007

Cited by 4 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Biomarkers for gene therapy clinical trials of lysosomal storage disorders;Molecular Therapy;2024-09

2. Real-world pharmacovigilance analysis of galsulfase: a study based on the FDA adverse event reporting system (FAERS) database;Frontiers in Pharmacology;2024-08-05

3. Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII;Molecular Genetics and Metabolism;2022-05

4. Update of treatment for mucopolysaccharidosis type III (sanfilippo syndrome);European Journal of Pharmacology;2020-12