Mini-COMET study: Safety, biomarker, and efficacy data after avalglucosidase alfa dosing for ≥ 97 weeks in participants with infantile-onset pompe disease (IOPD) previously treated with alglucosidase alfa who had demonstrated clinical decline

Authors
Category Primary study
JournalMolecular Genetics and Metabolism
Year 2022
This article has no abstract
Epistemonikos ID: bd3a1fac182e9cb47e702e5ca491f3eb02127477
First added on: Feb 13, 2025