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佩格瓦利酶治疗使成年人患有基尿症的血液神经递质代谢物正常化

Monika A Sigg1, Christopher Wilson2, Gillian E Clague1

  • 1BioMarin Pharmaceutical Inc., Novato, CA, USA.

Molecular genetics and metabolism
|October 10, 2024
PubMed
概括

基尿症 (PKU) 患者用佩格瓦里酶治疗使神经递质代谢物水平正常化. 这种酶疗法改善了血液代谢物水平,与PKU患者的氨酸 (Phe) 降低相关.

关键词:
生物标志物 生物标志物神经递质代谢物的代谢物.神经递质是神经递质中的一种.佩格瓦里亚斯 (Pegvaliase) 是一个氨酸氨酸是一种氨酸.基尿症 基尿症

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