Current Issues and Emerging Advances in the Management of PKU

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Presentation transcript:

Current Issues and Emerging Advances in the Management of PKU

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.

Phenylalanine Pathway in Phenylketonuria

What Is Phenylketonuria?

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

Goal of Treatment Control Blood [Phe]

Understanding the Basis of Nutritional Management of PKU

Dietary Management of PKU

PKU Diet Compliance Barriers

Treatment Strategies for Adults With PKU

Maternal PKU

Adjunct Pharmacotherapy Sapropterin Dihydrochloride

Adjunct Therapy Large Neutral Amino Acids

Pegvaliase What Is It and How Does It Work?

Pegvaliase PRISM-2 Study Results

Concluding Remarks and Looking Ahead

Abbreviations