Publication:
Vestronidase alfa for the treatment of Mucopolysaccharidosis VII (MPS VII): Updated results from a novel, longitudinal, multicenter disease monitoring program

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dc.contributor.authorGuigliani, Roberto
dc.contributor.authorLopez, Antonio Gonzalez-Meneses
dc.contributor.authorScarpa, Maurizio
dc.contributor.authorLau, Heather
dc.contributor.authorZhang, Lin
dc.contributor.authorMalkus, Betsy
dc.contributor.authorMarsden, Deborah
dc.contributor.authorMerritt, J. Lawrence, II
dc.contributor.authoraffiliation[Guigliani, Roberto] Univ Fed Rio Grande do Sul, Med Genet Serv HCPA, Dep Genet, Porto Alegre, RS, Brazil
dc.contributor.authoraffiliation[Guigliani, Roberto] INAGEMP, Porto Alegre, RS, Brazil
dc.contributor.authoraffiliation[Lopez, Antonio Gonzalez-Meneses] Hosp Univ Virgen del Rocio, Seville, Spain
dc.contributor.authoraffiliation[Scarpa, Maurizio] Univ Hosp Udine, Reg Coordinator Ctr Rare Dis, Udine, Italy
dc.contributor.authoraffiliation[Lau, Heather] Ultragenyx Pharmaceut Inc, Novato, CA USA
dc.contributor.authoraffiliation[Zhang, Lin] Ultragenyx Pharmaceut Inc, Novato, CA USA
dc.contributor.authoraffiliation[Malkus, Betsy] Ultragenyx Pharmaceut Inc, Novato, CA USA
dc.contributor.authoraffiliation[Marsden, Deborah] Ultragenyx Pharmaceut Inc, Novato, CA USA
dc.contributor.authoraffiliation[Merritt, J. Lawrence, II] Ultragenyx Pharmaceut Inc, Novato, CA USA
dc.date.accessioned2023-05-03T15:00:39Z
dc.date.available2023-05-03T15:00:39Z
dc.date.issued2022-03-21
dc.identifier.doi10.1016/j.gim.2022.01.202
dc.identifier.essn1530-0366
dc.identifier.issn1098-3600
dc.identifier.unpaywallURLhttp://www.gimjournal.org/article/S1098360022002192/pdf
dc.identifier.urihttp://hdl.handle.net/10668/22252
dc.identifier.wosID788465500155
dc.issue.number3
dc.issue.numberS
dc.journal.titleGenetics in medicine
dc.journal.titleabbreviationGenet. med.
dc.language.isoen
dc.organizationHospital Universitario Virgen del Rocío
dc.page.numberS101-S101
dc.publisherElsevier science inc
dc.rights.accessRightsopen access
dc.titleVestronidase alfa for the treatment of Mucopolysaccharidosis VII (MPS VII): Updated results from a novel, longitudinal, multicenter disease monitoring program
dc.typeconference output
dc.type.hasVersionVoR
dc.volume.number24
dc.wostypeMeeting Abstract
dspace.entity.typePublication

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