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Advances in gene therapy for phenylketonuria (PKU)

Advances in gene therapy for phenylketonuria (PKU). Cary O. Harding, MD Department of Molecular & Medical Genetics. Disclosures. BioMarin Corporation Funds for participation in clinical trials Sapropterin dihydrochloride rAvPAL-PEG National PKU Alliance Funds for PKU gene therapy research.

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Advances in gene therapy for phenylketonuria (PKU)