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...

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Detalles Bibliográficos
Autores principales: Tiraboschi, Gilles, Marchionni, David, Tuffal, Gilles, Fabre, David, Martinez, Jean-Marie, Haack, Kristina An, Miossec, Patrick, Kittner, Barbara, Daba, Nadia, Hurbin, Fabrice
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer US 2023
Materias:
Original Paper
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

Internet

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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