RT Journal Article
T1 Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.
A1 Jones, Simon
A1 Coker, Mahmut
A1 López, Antonio González-Meneses
A1 Sniadecki, Jennifer
A1 Mayhew, Jill
A1 Hensman, Pauline
A1 Jurecka, Agnieszka
K1 Enzyme replacement therapy
K1 MPS VII
K1 Mucopolysaccharidosis VII
K1 Phase 1/2
K1 Recombinant human β-glucuronidase
K1 Vestronidase alfa
AB 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.
SN 2214-4269
YR 2021
FD 2021-05-29
LK http://hdl.handle.net/10668/18006
UL http://hdl.handle.net/10668/18006
LA en
DS RISalud
RD Apr 11, 2025