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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Principais autores:	Timothy Nicholas Fazio, Louise Healy, Tim Heise, Anita Inwood, Catherine Manolikos, Yusof Rahman, Hans-Juergen Woerle, Christian J. Hendriksz
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2023-12-01
coleção:	Molecular Genetics and Metabolism Reports
Assuntos:	Phenylketonuria CDX-6114 Enzyme Cinnamic acid Phenylalanine hydroxylase
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2214426923000587
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