Real-world safety and effectiveness of velaglucerase alfa in pediatric patients with Gaucher disease younger than 4 years of age: A combined retrospective and prospective cohort study

Saved in:
Bibliographic Details
Published in:Molecular genetics and metabolism 2022-02, Vol.135 (2), p.S48-S49
Main Authors: Göker-Alpan, Ozlem, Friedman, Andrew, Limgala, Renuka, Ivanova, Margarita, Pathak, Ravi, Wright, Ekaterina
Format: Article
Language:English
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:
ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2021.11.116