biostudies-literatureUnknown28Jones SUltragenyx Pharmaceutical IncVestronidase 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.Molecular genetics and metabolism reports100774https://www.ebi.ac.uk/biostudies/studies/S-EPMC8178115biostudies-literatureOpen-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.PMC8178115Sniadecki JMayhew JHensman PCoker MLopez AGJurecka AJones SfalseOpen-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, 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.2021-01-01T00:00:00Z2021 Sep2024-11-12T14:22:53.636Z2022-02-10T15:21:24.13ZS-EPMC81781153413635710.1016/j.ymgmr.2021.100774