Population Pharmacokinetic Modeling and Determination of Individual Exposure to Avalglucosidase Alfa in Adolescent and Adult Patients With Late-Onset Pompe Disease: Analysis of Pooled Data From Phase I to III Clinical Trials

BACKGROUND: Pompe disease is a rare genetic disorder caused by a deficiency of a lysosomal enzyme called acid alpha-glucosidase and is classified into infantile and late-onset forms. Since 2006, an enzyme replacement therapy involving alglucosidase alfa has been available. In 2021, a new enzyme repl...

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Detalles Bibliográficos
Autores principales: Tuffal, Gilles, Tiraboschi, Gilles, Hurbin, Fabrice, Boittet, Pascale, Palmer, Rachel, Martinez, Jean-Marie, Fabre, David
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Therapeutic Drug Monitoring 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10497203/
https://www.ncbi.nlm.nih.gov/pubmed/37556417
http://dx.doi.org/10.1097/FTD.0000000000001086

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