Dr. Carey Harding presented the long-term efficacy and safety of pegvaliase treatment for phenylketonuria (PKU) based on the phase three PRISM trial data
1:24 - Study Design and Phases: Detailed description of the PRISM trial phases, including PRISM 1 and PRISM 2.
3:41 - Outcome Measures: Detailed outline of the outcome measures, including the incidence of adverse events, hypersensitivity reactions, and the primary efficacy of measuring blood phenylalanine concentrations.
5:08 - Adverse Events and Management: Discussion on the common adverse events, hypersensitivity reactions, and management strategies.
5:44 - Efficacy Outcomes: Presentation of the efficacy outcomes, including the proportion of individuals achieving treatment efficacy.
8:07 - Discussion and Conclusion: Summary of the study's findings, highlighting the importance of customized dosing and ongoing safety assessments.