{"title":"GC1111作为一种酶替代疗法,对既往未接受过治疗的II型粘多糖病(亨特综合征)患者进行的III期临床试验:双盲、随机、活性对照(第1部分)和开放标签、历史安慰剂对照(第2部分)研究","authors":"Dongkyu Jin, Jinsup Kim, A. Yang, SooKyung Shin","doi":"10.1016/j.ymgme.2023.107903","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":504050,"journal":{"name":"Molecular Genetics and Metabolism","volume":"53 ","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"A phase III clinical trial of GC1111 as an enzyme replacement therapy in previously untreated mucopolysaccharidosis type II (Hunter syndrome) patients: A double-blind, randomized, active-controlled (part 1) and open-labeled, historical placebo-controlled (part 2) study\",\"authors\":\"Dongkyu Jin, Jinsup Kim, A. Yang, SooKyung Shin\",\"doi\":\"10.1016/j.ymgme.2023.107903\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":504050,\"journal\":{\"name\":\"Molecular Genetics and Metabolism\",\"volume\":\"53 \",\"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.2023.107903\",\"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.2023.107903","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
A phase III clinical trial of GC1111 as an enzyme replacement therapy in previously untreated mucopolysaccharidosis type II (Hunter syndrome) patients: A double-blind, randomized, active-controlled (part 1) and open-labeled, historical placebo-controlled (part 2) study