T. Mozaffar, D. Bratkovic, B. Byrne, K. Claeys, Jordi Díaz-Manera, M. Dimachkie, Priya S. Kishnani, H. Kushlaf, Mark E. Roberts, Antonio Toscano, J. Castelli, F. Holdbrook, Sheela Sitaraman Das, Y. Wasfi, Benedikt Schoser
{"title":"在 PROPEL 项目中,对有 ERT 经验的成人晚发型庞贝氏症患者进行西帕糖苷酶α加米格鲁司特与阿糖苷酶α的效应大小分析","authors":"T. Mozaffar, D. Bratkovic, B. Byrne, K. Claeys, Jordi Díaz-Manera, M. Dimachkie, Priya S. Kishnani, H. Kushlaf, Mark E. Roberts, Antonio Toscano, J. Castelli, F. Holdbrook, Sheela Sitaraman Das, Y. Wasfi, Benedikt Schoser","doi":"10.1016/j.ymgme.2023.107971","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":504050,"journal":{"name":"Molecular Genetics and Metabolism","volume":"14 8","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL\",\"authors\":\"T. Mozaffar, D. Bratkovic, B. Byrne, K. Claeys, Jordi Díaz-Manera, M. Dimachkie, Priya S. Kishnani, H. Kushlaf, Mark E. Roberts, Antonio Toscano, J. Castelli, F. Holdbrook, Sheela Sitaraman Das, Y. Wasfi, Benedikt Schoser\",\"doi\":\"10.1016/j.ymgme.2023.107971\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":504050,\"journal\":{\"name\":\"Molecular Genetics and Metabolism\",\"volume\":\"14 8\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2024-02-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Molecular Genetics and Metabolism\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1016/j.ymgme.2023.107971\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Genetics and Metabolism","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.ymgme.2023.107971","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL