Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial
Journal
JAMA neurology
Journal Volume
80
Journal Issue
6
Date Issued
2023-06-01
Author(s)
Kishnani, Priya S
Diaz-Manera, Jordi
Toscano, Antonio
Clemens, Paula R
Ladha, Shafeeq
Berger, Kenneth I
Kushlaf, Hani
Straub, Volker
Carvalho, Gerson
Mozaffar, Tahseen
Roberts, Mark
Attarian, Shahram
Choi, Young-Chul
Day, John W
Erdem-Ozdamar, Sevim
Illarioshkin, Sergey
Goker-Alpan, Ozlem
Kostera-Pruszczyk, Anna
van der Ploeg, Ans T
An Haack, Kristina
Huynh-Ba, Olivier
Tammireddy, Swathi
Thibault, Nathan
Zhou, Tianyue
Dimachkie, Mazen M
Schoser, Benedikt
Abstract
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.
SDGs
Type
journal article