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Improvements in blood phenylalanine and health-related quality of life outcomes among adults with PKU receiving pegvaliase in the OPAL study

Publication summary

Description

OPAL is an ongoing, Phase 4, multicentre, observational study assessing the real-world safety and efficacy of pegvaliase in adults with PKU from the US, Germany and Italy. These results are from a second interim analysis.

Results showed that reductions in blood Phe levels in participants treated with pegvaliase were consistent with the Phase 3 PRISM clinical trial programme. At Week 96, PKU-QoL and PKU-SSIS scores improved. Final results from the OPAL study will further advance the understanding of the impact of pegvaliase treatment in routine clinical practice

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References:

McNutt M, Sazova O, Karimi M, Schwartz NRM, Rutsch F, Muntau AC. Genetics in Medicine Open. 2025. 102846.