TY  - CONF
AU  - Guigliani, Roberto
AU  - Lopez, Antonio Gonzalez-Meneses
AU  - Scarpa, Maurizio
AU  - Lau, Heather
AU  - Zhang, Lin
AU  - Malkus, Betsy
AU  - Marsden, Deborah
AU  - Merritt, J. Lawrence, II
PY  - 2022
DO  - 10.1016/j.gim.2022.01.202
SN  - 1098-3600
UR  - http://hdl.handle.net/10668/22252
T2  - Genetics in medicine
LA  - en
PB  - Elsevier science inc
TI  - Vestronidase alfa for the treatment of Mucopolysaccharidosis VII (MPS VII): Updated results from a novel, longitudinal, multicenter disease monitoring program
TY  - conference output
VL  - 24
ER  -