Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8342980 | Molecular Genetics and Metabolism | 2018 | 26 Pages |
Abstract
Mean blood Phe reduction was sustained in the pegvaliase group, while placebo groups had mean blood Phe concentration increase toward pre-treatment baseline levels. Results from this study confirmed the efficacy of pegvaliase in maintaining reduced blood Phe concentrations with a manageable safety profile for most participants.
Keywords
MMRMANCOVACANTABNIAID/FAANCTCAEMedDRAACMGCambridge Neuropsychological Test Automated BatterymITTmodified intent-to-treatanalysis of covarianceHAEMedical Dictionary for Regulatory Activitiesadverse eventconfidence intervalCommon Terminology Criteria for Adverse EventsAmerican College of Medical Genetics and GenomicsLeast squares
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Authors
Cary O. Harding, R. Stephen Amato, Mary Stuy, Nicola Longo, Barbara K. Burton, John Posner, Haoling H. Weng, Markus Merilainen, Zhonghua Gu, Joy Jiang, Jerry Vockley, PRISM-2 Investigators PRISM-2 Investigators,