Jones, SimonCoker, MahmutLópez, Antonio González-MenesesSniadecki, JenniferMayhew, JillHensman, PaulineJurecka, Agnieszka2023-02-092023-02-092021-05-292214-4269http://hdl.handle.net/10668/18006Vestronidase 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.enAttribution 4.0 Internationalhttp://creativecommons.org/licenses/by/4.0/Enzyme replacement therapyMPS VIIMucopolysaccharidosis VIIPhase 1/2Recombinant human β-glucuronidaseVestronidase alfaresearch article34136357open access10.1016/j.ymgmr.2021.100774PMC8178115https://doi.org/10.1016/j.ymgmr.2021.100774https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8178115/pdf