Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
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 JAMA neurology（2023）
摘要
ClinicalTrials.gov Identifier: NCT02782741.
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