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Open-Label Phase 1/2 Study of Vestronidase Alfa for Mucopolysaccharidosis VII

key information

source: Molecular genetics and metabolism reports

year: 2021

authors: Jones S,Coker M,López AG,Sniadecki J,Mayhew J,Hensman P,Jurecka A

summary/abstract:

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.

organization: Manchester University Hospitals NHS Foundation Trust, St Mary's Hospital, Manchester, UK.

DOI: 10.1016/j.ymgmr.2021.100774

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