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

Mucopolysaccharidosis (MPS) VII is an ultra-rare, autosomal-recessive, metabolic disease caused by a deficiency of β-glucuronidase, a lysosomal enzyme that hydrolyzes glycosaminoglycans (GAGs), including dermatan sulfate (DS), chondroitin sulfate, and heparan sulfate (HS). β-glucuronidase deficiency...

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Bibliographic Details
Published in:	Molecular genetics and metabolism Vol. 136; no. 1; pp. 28 - 37
Main Authors:	Lau, Heather A., Viskochil, David, Tanpaiboon, Pranoot, Lopez, Antonio Gonzalez-Meneses, Martins, Esmeralda, Taylor, Julie, Malkus, Betsy, Zhang, Lin, Jurecka, Agnieszka, Marsden, Deborah
Format:	Journal Article
Language:	English
Published:	United States Elsevier Inc 01.05.2022
Subjects:	Growth Hepatosplenomegaly Mucopolysaccharidosis VII Pediatric patients Urinary glycosaminoglycan Vestronidase alfa NRE Urinary glycosaminoglycan CPAP suppl O2 Growth ERT LS Mucopolysaccharidosis VII CGI-C HS DS LC-MS/MS MPS SD SE ULN Vestronidase alfa NIHF Hepatosplenomegaly SpO2 h BiPAP Pediatric patients GAG m uGAG CS TEAE MS-DS SAE QOW IAR ADA
ISSN:	1096-7192, 1096-7206, 1096-7206
Online Access:	Get full text
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