PTC Therapeutics reports new Sephience PKU trial and real-world data showing sustained Phe control, diet liberalization
PTCT•Sephience PKU data slated for SSIEM presentation
PTC Therapeutics outlined new Sephience (sepiapterin) trial and real-world analyses slated for presentation at the SSIEM meeting on Aug. 25-28.
- Upcoming presentations point to sustained metabolic control in PKU, with many patients reaching normal-range blood phenylalanine quickly.
- Data also suggest meaningful diet liberalization while maintaining recommended control, including in patients with more severe or historically less responsive disease.
- Safety findings remained consistent with prior studies, supporting broader use across patient subgroups.
