Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.,JAMA Neurology

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Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
JAMA Neurology ( IF 20.4 ) Pub Date : 2023-04-10 , DOI: 10.1001/jamaneurol.2023.0552
Priya S Kishnani, Jordi Diaz-Manera, Antonio Toscano, Paula R Clemens, Shafeeq Ladha, Kenneth I Berger, Hani Kushlaf, Volker Straub, Gerson Carvalho, Tahseen Mozaffar, Mark Roberts, Shahram Attarian, Yin-Hsiu Chien, Young-Chul Choi, John W Day, Sevim Erdem-Ozdamar, Sergey Illarioshkin, Ozlem Goker-Alpan, Anna Kostera-Pruszczyk, Ans T van der Ploeg, Kristina An Haack, Olivier Huynh-Ba, Swathi Tammireddy, Nathan Thibault, Tianyue Zhou, Mazen M Dimachkie, Benedikt Schoser

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.

中文翻译：

Avalglucosidase Alfa 治疗迟发性庞贝病患者 97 周后的疗效和安全性：一项 3 期随机临床试验。

在先前报道的使用 neoGAA 与 rhGAA 进行的比较酶替代试验 (COMET) 试验中，与阿糖苷酶 alfa 相比，阿瓦尔葡萄糖苷酶 alfa 治疗 49 周显示直立用力肺活量 (FVC) 预测百分比和 6 分钟步行试验 (6MWT) 具有临床意义的改善.

更新日期：2023-04-10

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