TY  - JOUR
AU  - Jones, Simon
AU  - Coker, Mahmut
AU  - López, Antonio González-Meneses
AU  - Sniadecki, Jennifer
AU  - Mayhew, Jill
AU  - Hensman, Pauline
AU  - Jurecka, Agnieszka
PY  - 2021
DO  - 10.1016/j.ymgmr.2021.100774
SN  - 2214-4269
UR  - http://hdl.handle.net/10668/18006
T2  - Molecular genetics and metabolism reports
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...
LA  - en
KW  - Enzyme replacement therapy
KW  - MPS VII
KW  - Mucopolysaccharidosis VII
KW  - Phase 1/2
KW  - Recombinant human β-glucuronidase
KW  - Vestronidase alfa
TI  - Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.
TY  - research article
VL  - 28
ER  -