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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Detalles Bibliográficos
Autores principales: Jones, Simon, Coker, Mahmut, López, Antonio González-Meneses, Sniadecki, Jennifer, Mayhew, Jill, Hensman, Pauline, Jurecka, Agnieszka
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Short Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8178115/
https://www.ncbi.nlm.nih.gov/pubmed/34136357
http://dx.doi.org/10.1016/j.ymgmr.2021.100774
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author Jones, Simon
Coker, Mahmut
López, Antonio González-Meneses
Sniadecki, Jennifer
Mayhew, Jill
Hensman, Pauline
Jurecka, Agnieszka
author_facet Jones, Simon
Coker, Mahmut
López, Antonio González-Meneses
Sniadecki, Jennifer
Mayhew, Jill
Hensman, Pauline
Jurecka, Agnieszka
author_sort Jones, Simon
collection PubMed
description 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, 4, and 2 mg/kg QOW; 8 weeks each), 36-week continuation (2 mg/kg), and long-term extension (4 mg/kg). Vestronidase alfa was well tolerated and led to dose-responsive, sustained reductions in urinary glycosaminoglycan excretion.
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spelling pubmed-81781152021-06-15 Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII Jones, Simon Coker, Mahmut López, Antonio González-Meneses Sniadecki, Jennifer Mayhew, Jill Hensman, Pauline Jurecka, Agnieszka Mol Genet Metab Rep Short Communication 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, 4, and 2 mg/kg QOW; 8 weeks each), 36-week continuation (2 mg/kg), and long-term extension (4 mg/kg). Vestronidase alfa was well tolerated and led to dose-responsive, sustained reductions in urinary glycosaminoglycan excretion. Elsevier 2021-05-29 /pmc/articles/PMC8178115/ /pubmed/34136357 http://dx.doi.org/10.1016/j.ymgmr.2021.100774 Text en © 2021 Published by Elsevier Inc. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Short Communication
Jones, Simon
Coker, Mahmut
López, Antonio González-Meneses
Sniadecki, Jennifer
Mayhew, Jill
Hensman, Pauline
Jurecka, Agnieszka
Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII
title Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII
title_full Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII
title_fullStr Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII
title_full_unstemmed Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII
title_short Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII
title_sort open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis vii
topic Short Communication
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8178115/
https://www.ncbi.nlm.nih.gov/pubmed/34136357
http://dx.doi.org/10.1016/j.ymgmr.2021.100774
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