Jones, SimonCoker, MahmutGonzález-Meneses López, AntonioSniadecki, JenniferMayhew, JillHensman, PaulineJurecka, Agnieszka2025-05-212025-05-212021-05-29Jones, S., Coker, M., González-Meneses López, A., Sniadecki, J., Mayhew, J., Hensman, P. y Jurecka, A. (2021). Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII. Molecular Genetics and Metabolism Reports, 28, 100774. https://doi.org/10.1016/j.ymgmr.2021.100774.2214-4269https://hdl.handle.net/11441/173076Vestronidase 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.application/pdf4 p.engAttribution 4.0 Internationalhttp://creativecommons.org/licenses/by/4.0/vestronidase alfarecombinant human β-glucuronidaseenzyme replacement therapymucopolysaccharidosis VIIMPS VIIphase 1/2info:eu-repo/semantics/articleinfo:eu-repo/semantics/openAccesshttps://doi.org/10.1016/j.ymgmr.2021.100774