Association of immune response with efficacy and safety outcomes in adults with phenylketonuria administered pegvaliase in phase 3 clinical trials.
EBioMedicine(2018)
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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