학술논문
Using a novel measuring device to objectively evaluate the clinical benefit on ptosis in infantile-onset Pompe disease (IOPD): Results from 145 weeks' avalglucosidase alfa dosing in Mini-COMET participants previously treated with alglucosidase alfa
Document Type
Abstract
Author
Kishnani, Priya S.; Weinert, Marguerite C.; Davison, James; Brassier, Anais; Broomfield, Alexander; Chien, Yin-Hsiu; Hahn, Sihoun; Kronn, David; Kumada, Satoko; Labarthe, François; Ohki, Hirotaka; Haack, Kristina An; Huynh-Ba, Olivier; Sparks, Susan; Tammireddy, Swathi; Zhou, Tianyue; Prakalapakorn, S. Grace
Source
In Molecular Genetics and Metabolism February 2024 141(2)
Subject
Language
ISSN
1096-7192