Long-Term Efficacy and Safety of Avalglucosidase Alfa Therapy in Patients With Late-Onset Pompe Disease
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JAMA Neurology
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial
JAMA Neurol 2023 Apr 10;[EPub Ahead of Print], PS Kishnani, J Diaz-Manera, A Toscano, PR Clemens, S Ladha, KI Berger, H Kushlaf, V Straub, G Carvalho, T Mozaffar, M Roberts, S Attarian, YH Chien, YC Choi, JW Day, S Erdem-Ozdamar, S Illarioshkin, O Goker-Alpan, A Kostera-Pruszczyk, AT van der Ploeg, K An Haack, O Huynh-Ba, S Tammireddy, N Thibault, T Zhou, MM Dimachkie, B SchoserFrom MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.