Jones Simon, Coker Mahmut, López Antonio González-Meneses, Sniadecki Jennifer, Mayhew Jill, Hensman Pauline, Jurecka Agnieszka
Manchester University Hospitals NHS Foundation Trust, St Mary's Hospital, Manchester, UK.
Ege University, Bornova, Izmir, Turkey.
Mol Genet Metab Rep. 2021 May 29;28:100774. doi: 10.1016/j.ymgmr.2021.100774. eCollection 2021 Sep.
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.
维司妥酶α是一种用于治疗黏多糖贮积症VII型（MPS VII）的酶替代疗法。在这项开放标签的1/2期研究中，三名MPS VII患者接受了每两周一次（QOW）静脉注射维司妥酶α，持续14周（2mg/kg），随后进行24周的强制剂量滴定（每两周一次，剂量分别为1、4和2mg/kg；各8周）、36周的持续治疗（2mg/kg）以及长期扩展治疗（4mg/kg）。维司妥酶α耐受性良好，可导致尿糖胺聚糖排泄量出现剂量依赖性的持续降低。
