Mucopolysaccharidosis type II sibling pairs study: A global retrospective chart review of effectiveness of idursulfase in infants and very young children with MPS II

{"title":"Mucopolysaccharidosis type II sibling pairs study: A global retrospective chart review of effectiveness of idursulfase in infants and very young children with MPS II","authors":"Barbara K. Burton , Bernd Schweikert , Olivia Okoli , Diana Sanchez-Hernandez , Jaco Botha , Jennifer Audi , Csaba Siffel , Olulade Ayodele","doi":"10.1016/j.ymgme.2024.108672","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108672"},"PeriodicalIF":3.7000,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular genetics and metabolism","FirstCategoryId":"99","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1096719224005560","RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}