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Perspective interview - Ania Muntau (PKU treatment in children aged under 4)

Perspective interview - Ania Muntau (PKU treatment in children aged under 4)

Muntau 150911

PKU treatment in children aged <4 years

1. What treatments are available for PKU in children aged <4 years?])

2. What are the disadvantages of a protein-restricted diet?

3. How well do parents and their children cope with the diet restrictions?

4. Is BH4 likely to be indicated for use in children aged <4 years in the future?

5. What are the potential advantages of BH4 cofactor treatment?

6. Are there any safety concerns of BH4 cofactor treatment in young children?

7. How do you see PKU treatment of children evolving in the future?

Ania Muntau

Inborn Errors of Metabolism (IEM) and of Molecular Pediatrics
Dr von Hauner Children's Hospital
Ludwig-Maximilians-University
Munich, Germany