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Future treatment strategies in phenylketonuria

Francjan J van Spronsen1, Gregory M Enns

  • 1Section of Metabolic Diseases, Beatrix Children's Hospital, University Medical Centre of Groningen, PO Box 30.001, 9700 RB, Groningen, The Netherlands. f.j.van.spronsen@bkk.umcq.nl

Molecular Genetics and Metabolism
|February 4, 2010
PubMed
Summary

Phenylketonuria (PKU) treatment prevents brain damage with early low-phenylalanine diets. New strategies like enzyme, gene, and cellular therapies aim to further improve outcomes for PKU patients facing dietary adherence and cognitive challenges.

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