Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial
| Autor: | Toscano, Antonio, Pollissard, Laurence, Msihid, Jérôme, van der Beek, Nadine, Kishnani, Priya S., Dimachkie, Mazen M., Berger, Kenneth I., DasMahapatra, Pronabesh, Thibault, Nathan, Hamed, Alaa, Zhou, Tianyue, Haack, Kristina An, Schoser, Benedikt |
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| Zdroj: | In Molecular Genetics and Metabolism February 2024 141(2) |
| Databáze: | ScienceDirect |
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