{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["28"],"submitter":["Jones S"],"funding":["Ultragenyx Pharmaceutical Inc"],"pubmed_abstract":["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."],"journal":["Molecular genetics and metabolism reports"],"pagination":["100774"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC8178115"],"repository":["biostudies-literature"],"pubmed_title":["Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII."],"pmcid":["PMC8178115"],"pubmed_authors":["Sniadecki J","Mayhew J","Hensman P","Coker M","Lopez AG","Jurecka A","Jones S"],"additional_accession":[]},"is_claimable":false,"name":"Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.","description":"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.","dates":{"release":"2021-01-01T00:00:00Z","publication":"2021 Sep","modification":"2024-11-12T14:22:53.636Z","creation":"2022-02-10T15:21:24.13Z"},"accession":"S-EPMC8178115","cross_references":{"pubmed":["34136357"],"doi":["10.1016/j.ymgmr.2021.100774"]}}