The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII-Reference-Cited by-同舟云学术

The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII

Published:2020-03 Issue:3 Volume:129 Page:219-227
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Wang Raymond Y.,da Silva Franco José Francisco,López-Valdez Jaime,Martins Esmeralda,Sutton Vernon Reid,Whitley Chester B.,Zhang Lin,Cimms Tricia,Marsden Deborah,Jurecka Agnieszka,Harmatz Paul

Funder

Ultragenyx Pharmaceutical Inc

Ultragenyx Pharmaceutical

Publisher

Elsevier BV

Subject

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

Reference41 articles.

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3. Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly Syndrome);Tomatsu;HumMutat,2009

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

5. Quantitative clinical characteristics of 53 patients with MPS VII: a cross-sectional analysis;Zielonka;Genet. Med.,2017

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