https://scholars.lib.ntu.edu.tw/handle/123456789/636688
標題: | Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial | 作者: | Kishnani, Priya S Diaz-Manera, Jordi Toscano, Antonio Clemens, Paula R Ladha, Shafeeq Berger, Kenneth I Kushlaf, Hani Straub, Volker Carvalho, Gerson Mozaffar, Tahseen Roberts, Mark Attarian, Shahram YIN-HSIU CHIEN Choi, Young-Chul Day, John W Erdem-Ozdamar, Sevim Illarioshkin, Sergey Goker-Alpan, Ozlem Kostera-Pruszczyk, Anna van der Ploeg, Ans T An Haack, Kristina Huynh-Ba, Olivier Tammireddy, Swathi Thibault, Nathan Zhou, Tianyue Dimachkie, Mazen M Schoser, Benedikt |
公開日期: | 1-六月-2023 | 卷: | 80 | 期: | 6 | 來源出版物: | JAMA neurology | 摘要: | In the previously reported Comparative Enzyme Replacement Trial With neoGAA Versus rhGAA (COMET) trial, avalglucosidase alfa treatment for 49 weeks showed clinically meaningful improvements in upright forced vital capacity (FVC) percent predicted and 6-minute walk test (6MWT) compared with alglucosidase alfa. |
URI: | https://scholars.lib.ntu.edu.tw/handle/123456789/636688 | ISSN: | 21686149 | DOI: | 10.1001/jamaneurol.2023.0552 |
顯示於: | 醫學系 |
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