학술논문
Efficacy and Safety of Avalglucosidase Alfa in Patients with Late-Onset Pompe Disease after 97 Weeks: A Phase 3 Randomized Clinical Trial
Document Type
Article
Author
Kishnani, P.S.; Diaz-Manera, J.; Straub, V.; Toscano, A.; Clemens, P.R.; Ladha, S.; Berger, K.I.; Kushlaf, H.; Carvalho, G.; Mozaffar, T.; Roberts, M.; Attarian, S.; Chien, Y.-H.; Choi, Y.-C.; Day, J.W.; Erdem-Ozdamar, S.; Illarioshkin, S.; Goker-Alpan, O.; Kostera-Pruszczyk, A.; Van Der Ploeg, A.T.; An Haack, K.; Huynh-Ba, O.; Tammireddy, S.; Thibault, N.; Zhou, T.; Dimachkie, M.M.; Schoser, B.
Source
In: JAMA Neurology . (JAMA Neurology, 12 June 2023, 80(6):558-567)
Subject
Language
English
ISSN
21686157
21686149
21686149