1. Current Issues and Emerging Advances in the Management of PKU

3. This program will include a discussion of investigational agents not yet approved by the FDA for use in the United States. There will also be a discussion of data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.

4. Phenylalanine Pathway in Phenylketonuria

5. What Is Phenylketonuria?

6. Loss of Metabolic Control Possible Complications of Long-Term Elevated Blood [Phe]

7. Goal of Treatment Control Blood [Phe]

8. Understanding the Basis of Nutritional Management of PKU

9. Dietary Management of PKU

10. PKU Diet Compliance Barriers

11. Treatment Strategies for Adults With PKU

12. Maternal PKU

13. Adjunct Pharmacotherapy Sapropterin Dihydrochloride

14. Adjunct Therapy Large Neutral Amino Acids

15. Pegvaliase What Is It and How Does It Work?

16. Pegvaliase PRISM-2 Study Results

17. Concluding Remarks and Looking Ahead

18. Abbreviations