https://scholars.lib.ntu.edu.tw/handle/123456789/627122
標題: | Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report | 作者: | Kishnani, Priya S Kronn, David Brassier, Anaïs Broomfield, Alexander Davison, James Hahn, Si Houn Kumada, Satoko Labarthe, François Ohki, Hirotaka Pichard, Samia Prakalapakorn, S Grace Haack, Kristina An Kittner, Barbara Meng, Xianzhang Sparks, Susan Wilson, Catherine Zaher, Atef YIN-HSIU CHIEN |
關鍵字: | Acid α-glucosidase (GAA) deficiency; Alglucosidase alfa; Avalglucosidase alfa; Enzyme replacement therapy; Infantile-onset Pompe disease | 公開日期: | 20-十二月-2022 | 來源出版物: | Genetics in medicine : official journal of the American College of Medical Genetics | 摘要: | Mini-COMET (NCT03019406; Sanofi) is a phase 2, open-label, ascending-dose, 3-cohort study, evaluating avalglucosidase alfa safety, pharmacokinetics, and efficacy in individuals with infantile-onset Pompe disease aged <18 years who previously received alglucosidase alfa and showed clinical decline (cohorts 1 and 2) or suboptimal response (cohort 3). |
URI: | https://scholars.lib.ntu.edu.tw/handle/123456789/627122 | ISSN: | 10983600 | DOI: | 10.1016/j.gim.2022.10.010 |
顯示於: | 醫學系 |
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