Safety of arimoclomol in a pediatric sub-study of Niemann-Pick disease type C patients aged 6 to 24 months at study enrolment

{"title":"Safety of arimoclomol in a pediatric sub-study of Niemann-Pick disease type C patients aged 6 to 24 months at study enrolment","authors":"Christine I. Dali , Malene Cording , Stephanie Grunewald , Sabine Groenborg , Marc Patterson , Eugen Mengel","doi":"10.1016/j.ymgme.2024.108692","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108692"},"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/S1096719224005766","RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}