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Population pharmacokinetic modeling and dosing simulation of avalglucosidase alfa for selecting alternative dosing regimen in pediatric patients with late-onset pompe disease
Avalglucosidase alfa (AVAL) was approved in the United States (2021) for patients with late-onset Pompe disease (LOPD), aged ≥ 1 year. In the present study, pharmacokinetic (PK) simulations were conducted to propose alternative dosing regimens for pediatric LOPD patients based on a bodyweight cut-of...
Autores principales: | Tiraboschi, Gilles, Marchionni, David, Tuffal, Gilles, Fabre, David, Martinez, Jean-Marie, Haack, Kristina An, Miossec, Patrick, Kittner, Barbara, Daba, Nadia, Hurbin, Fabrice |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer US
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10673948/ https://www.ncbi.nlm.nih.gov/pubmed/37535240 http://dx.doi.org/10.1007/s10928-023-09874-8 |
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