Kylie Gray, Michael DiGruccio, Jonathan Roberts, Vaughn Weaver, Riley Marcinczyk, Linda Lyons, Udayanga S Wanninayake, Shou-nian Liu, N. Brignol, Osman Sheikh, Clarissa Booth, Steven B. Ortmeier, Katherine White, Hung Do, Russell Gotshall
{"title":"M021 (rhGAA)具有独特的糖基化特征,能更有效地减少糖原,可用于替代庞贝 ERT 给药策略","authors":"Kylie Gray, Michael DiGruccio, Jonathan Roberts, Vaughn Weaver, Riley Marcinczyk, Linda Lyons, Udayanga S Wanninayake, Shou-nian Liu, N. Brignol, Osman Sheikh, Clarissa Booth, Steven B. Ortmeier, Katherine White, Hung Do, Russell Gotshall","doi":"10.1016/j.ymgme.2023.107861","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":504050,"journal":{"name":"Molecular Genetics and Metabolism","volume":"11 9","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"M021 (rhGAA) has a unique glycosylation profile which enables more efficient glycogen reduction and may allow for alternative Pompe ERT dosing strategies\",\"authors\":\"Kylie Gray, Michael DiGruccio, Jonathan Roberts, Vaughn Weaver, Riley Marcinczyk, Linda Lyons, Udayanga S Wanninayake, Shou-nian Liu, N. Brignol, Osman Sheikh, Clarissa Booth, Steven B. Ortmeier, Katherine White, Hung Do, Russell Gotshall\",\"doi\":\"10.1016/j.ymgme.2023.107861\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":504050,\"journal\":{\"name\":\"Molecular Genetics and Metabolism\",\"volume\":\"11 9\",\"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.2023.107861\",\"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.2023.107861","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
M021 (rhGAA) has a unique glycosylation profile which enables more efficient glycogen reduction and may allow for alternative Pompe ERT dosing strategies