Prospective observational study to assess the long-term safety of olipudase alfa effect in pediatric patients less than 2 years of age with acid sphingomyelinase deficiency: Study design

{"title":"Prospective observational study to assess the long-term safety of olipudase alfa effect in pediatric patients less than 2 years of age with acid sphingomyelinase deficiency: Study design","authors":"Pablo Bianculli , Sefika Uslu Cil , Daniel Lewi , Kathleen Coolidge , Femida Gwadry-Sridhar , Antonio Oliveira-dos-Santos","doi":"10.1016/j.ymgme.2024.108660","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108660"},"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/S1096719224005444","RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}