%0 Journal Article
%A Jones, Simon
%A Coker, Mahmut
%A López, Antonio González-Meneses
%A Sniadecki, Jennifer
%A Mayhew, Jill
%A Hensman, Pauline
%A Jurecka, Agnieszka
%T Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.
%D 2021
%@ 2214-4269
%U http://hdl.handle.net/10668/18006
%X 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.
%K Enzyme replacement therapy
%K MPS VII
%K Mucopolysaccharidosis VII
%K Phase 1/2
%K Recombinant human β-glucuronidase
%K Vestronidase alfa
%~