학술논문
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
Document Type
Article
Author
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; Chien, Yin-Hsiu
Source
In Genetics in Medicine February 2023 25(2)
Subject
Language
ISSN
1098-3600