NPKUA’s sixth annual Scientific Conference took place February 26–28, 2026, in New Orleans, LA, bringing together leaders from across the PKU community. More than 100 invited healthcare providers, researchers, industry partners and regulatory representatives gathered to discuss the challenges of conducting clinical trials in PKU and together explored ways to improve them.
Key topics discussed included:
• Next steps after TruePKU and how our PKU community voices can improve future clinical trials;
• Continue the work towards achieving diet liberalization and other quality-of-life outcomes on the labels of new PKU medications to expand their indication beyond lowering blood Phe;
• Improve clinical trial designs for developing PKU investigational drugs that can determine safety and efficacy while minimizing patient burden.
The conversations were collaborative and solution-oriented, reflecting a shared commitment to strengthening clinical trial design and ensuring research efforts better serve individuals and families living with PKU.
Thank you to all who contributed their time and expertise to these important discussions, and to our industry sponsors whose support made the conference possible! We were proud to bring the PKU scientific community together again to move progress forward in the management of those living with PKU.
