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

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dc.contributor.authorJones, Simon
dc.contributor.authorCoker, Mahmut
dc.contributor.authorLópez, Antonio González-Meneses
dc.contributor.authorSniadecki, Jennifer
dc.contributor.authorMayhew, Jill
dc.contributor.authorHensman, Pauline
dc.contributor.authorJurecka, Agnieszka
dc.date.accessioned2023-02-09T11:40:36Z
dc.date.available2023-02-09T11:40:36Z
dc.date.issued2021-05-29
dc.description.abstractVestronidase 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.
dc.identifier.doi10.1016/j.ymgmr.2021.100774
dc.identifier.issn2214-4269
dc.identifier.pmcPMC8178115
dc.identifier.pmid34136357
dc.identifier.pubmedURLhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC8178115/pdf
dc.identifier.unpaywallURLhttps://doi.org/10.1016/j.ymgmr.2021.100774
dc.identifier.urihttp://hdl.handle.net/10668/18006
dc.journal.titleMolecular genetics and metabolism reports
dc.journal.titleabbreviationMol Genet Metab Rep
dc.language.isoen
dc.organizationHospital Universitario Virgen del Rocío
dc.page.number100774
dc.pubmedtypeJournal Article
dc.rightsAttribution 4.0 International
dc.rights.accessRightsopen access
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/
dc.subjectEnzyme replacement therapy
dc.subjectMPS VII
dc.subjectMucopolysaccharidosis VII
dc.subjectPhase 1/2
dc.subjectRecombinant human β-glucuronidase
dc.subjectVestronidase alfa
dc.titleOpen-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.
dc.typeresearch article
dc.type.hasVersionVoR
dc.volume.number28
dspace.entity.typePublication

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