Long‐Term Safety and Efficacy of Pegvaliase in Japanese Adults With Phenylketonuria: Final Results of a Phase III Trial

ABSTRACT Phenylketonuria (PKU) is an inborn error of metabolism leading to phenylalanine (Phe) accumulation and consequent neurological, neurocognitive, and psychiatric symptoms. Pegvaliase, a pegylated recombinant phenylalanine ammonia lyase that metabolizes Phe, effectively reduced blood Phe in ph...

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Главные авторы:	Yoko Nakajima, Mika Ishige, Tetsuya Ito, Takashi Hamazaki, Mitsuhiro Kuwahara, Lawrence Lee, Haruo Shintaku
Формат:	Artigo
Язык:	Inglês
Опубликовано:	Wiley 2026-05-01
Серии:	JIMD Reports
Предметы:	diet Japan pegvaliase PEGylated phenylalanine ammonia lyase phenylalanine hydroxylase deficiency phenylketonuria
Online-ссылка:	https://doi.org/10.1002/jmd2.70084
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