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
| Autor: | Kronn, David, Kishnani, Priya, Davison, James, Brassier, Anais, Broomfield, Alexander, Hahn, Sihoun, Kumada, Satoko, Labarthe, François, Ohki, Hirotaka, Haack, Kristina An, Sparks, Susan, Tammireddy, Swathi, Zaher, Atef, Zhou, Tianyue, Chien, Yin-Hsiu |
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| Zdroj: | In Molecular Genetics and Metabolism February 2024 141(2) |
| Databáze: | ScienceDirect |
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