Association of immune response with efficacy and safety outcomes in adults with phenylketonuria administered pegvaliase in phase 3 clinical trials.

Soumi Gupta,Kelly Lau,Cary O Harding,Gillian Shepherd,Ryan Boyer,John P Atkinson,Vijaya Knight,Joy Olbertz,Kevin Larimore,Zhonghu Gu,Mingjin Li,Orli Rosen,Stephen J Zoog,Haoling H Weng,Becky Schweighardt

EBioMedicine（2018）

引用 51|浏览30

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摘要

Despite a universal ADA response post-pegvaliase administration, adult patients with PKU achieved substantial and sustained blood Phe reductions with a manageable safety profile. FUND: BioMarin Pharmaceutical Inc.

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关键词

Enzyme replacement therapy,Antidrug antibody,Circulating immune complex,Hypersensitivity,Phenylalanine

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