Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII-Reference-Cited by-同舟云学术

Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII

Published:2022-05 Issue:1 Volume:136 Page:28-37
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Lau Heather A.,Viskochil David,Tanpaiboon Pranoot,Lopez Antonio Gonzalez-Meneses,Martins Esmeralda,Taylor Julie,Malkus Betsy,Zhang Lin,Jurecka Agnieszka,Marsden Deborah

Publisher

Elsevier BV

Subject

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

Reference35 articles.

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3. Vestronidase alfa: a review in mucopolysaccharidosis;McCafferty,2019

4. Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly syndrome);Tomatsu;Hum. Mutat.,2009

5. Association between mucopolysaccharidosis type VII and hydrops fetalis;Holtz;Ultrasound Obstet. Gynecol.,2020

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3. Dose-dependent effects of enzyme replacement therapy on skeletal disease progression in mucopolysaccharidosis VII dogs;Molecular Therapy - Methods & Clinical Development;2023-03

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