OP016: Mini-COMET: Safety and efficacy of ≥97 weeks’ avalglucosidase alfa in infantile-onset Pompe disease participants previously treated with alglucosidase alfa
| Autor: | Kronn, David, Davison, James, Brassier, Anaïs, Broomfield, Alexander, Hahn, Si Houn, Kumada, Satoko, Labarthe, François, Ohki, Hirotaka, Prakalapakorn, S. Grace, Haack, Kristina An, Meng, Xianzhang, Sparks, Susan, Tammireddy, Swathi, Wilson, Catherine, Zaher, Atef, Zhou, Tianyue, Chien, Yin-Hsiu, Kishnani, Priya, On behalf of the Mini-COMET investigators |
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| Zdroj: | In Genetics in Medicine March 2022 24(3) Supplement:S348-S349 |
| Databáze: | ScienceDirect |
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