학술논문
Mini-COMET study: Safety and efficacy data after avalglucosidase alfa dosing for ≥ 145 weeks in participants with infantile-onset Pompe disease (IOPD) who had demonstrated clinical decline or sub-optimal response while receiving alglucosidase alfa
Document Type
Abstract
Author
Source
In Molecular Genetics and Metabolism February 2024 141(2)
Subject
Language
ISSN
1096-7192