Pharmacodynamics, safety, tolerability and pharmacokinetics of a single oral dose of an engineered phenylalanine ammonia-lyase in patients with phenylketonuria

The cornerstone treatment of hyperphenylalaninemia (HPA) and phenylketonuria (PKU) is a lifelong low-protein diet with phenylalanine (Phe) free L-amino acid supplements. However, the PKU diet has significant shortcomings, and there is a clinically unmet need for new therapeutics to improve patient o...

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Detalles Bibliográficos
Autores principales: Fazio, Timothy Nicholas, Healy, Louise, Heise, Tim, Inwood, Anita, Manolikos, Catherine, Rahman, Yusof, Woerle, Hans-Juergen, Hendriksz, Christian J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10694774/
http://dx.doi.org/10.1016/j.ymgmr.2023.101012

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10694774/
http://dx.doi.org/10.1016/j.ymgmr.2023.101012

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