P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe Registry

Bibliographic Details
Main Authors: Priya Kishnani, David Stockton, Andreas Hahn, Juan Llerena, Alexander Broomfield, Julie Batista, Meredith Foster, Kathryn Wilson, Susan Sparks, Hannerieke van den Hout, Yin-Hsiu Chien
Format: Article
Language:English
Published: Elsevier 2024-01-01
Series:Genetics in Medicine Open
Online Access:http://www.sciencedirect.com/science/article/pii/S2949774424000529