Switching treatment to cipaglucosidase alfa plus miglustat positively affects motor function and quality of life in patients with late-onset Pompe disease

Switching treatment to cipaglucosidase alfa plus miglustat positively affects motor function and quality of life in patients with late-onset Pompe disease

Saved in:
Bibliographic Details
Published in:Molecular genetics and metabolism Vol. 141; no. 2; p. 108059
Main Authors: Toscano, Antonio, Byrne, Barry J., Claeys, Kristl G., Diaz-Manera, Jordi, Dimachkie, Mazen M., Kishnani, Priya S., Kushlaf, Hani, Mozaffar, Tahseen, Roberts, Mark E., Hummel, Noemi, Holdbrook, Fred K., Shohet, Simon, Wasfi, Yasmine, Schoser, Benedikt
Format: Journal Article
Language:English
Published: Elsevier Inc 01-02-2024
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2023.108059