Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII-Reference-Cited by-同舟云学术

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Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII

Published:2020-05 Issue:1 Volume:130 Page:65-76
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Cadaoas Jaclyn,Boyle Gabrielle,Jungles Steven,Cullen Sean,Vellard Michel,Grubb Jeffrey H.,Jurecka Agnieszka,Sly William,Kakkis Emil

Funder

Ultragenyx Pharmaceutical Inc.

Publisher

Elsevier BV

Subject

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

Reference27 articles.

1. Enzymatic formation of monosaccharides from hyaluronate;Linker;J. Biol. Chem.,1955

2. Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis;Sly;J.Pediatr.,1973

3. Clinical course of sly syndrome (mucopolysaccharidosis type VII);Montano;J. Med. Genet.,2016

4. Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival;Vogler;Pediatr. Res.,1996

5. Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII;Vogler;Proc. Natl. Acad. Sci. U. S. A.,2005

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