Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII

Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII

Vestronidase alfa is an enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII). In this open-label, phase 1/2 study, three subjects with MPS VII received intravenous vestronidase alfa administered every other week (QOW) for 14 weeks (2 mg/kg), followed by 24-week forced-dose titration (1...

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Bibliographic Details
Main Authors: Simon Jones, Mahmut Coker, Antonio González-Meneses López, Jennifer Sniadecki, Jill Mayhew, Pauline Hensman, Agnieszka Jurecka
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Vestronidase alfa
Recombinant human β-glucuronidase
Enzyme replacement therapy
Mucopolysaccharidosis VII
MPS VII
Phase 1/2
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426921000689

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http://www.sciencedirect.com/science/article/pii/S2214426921000689

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