Starting the conversation on gene therapy for phenylketonuria: Current perspectives of patients, caregivers, and advocates.

Mol Genet Metab Rep · 2022 · PMC9248220 · PMID 35782620

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an apparent trendno p-value reported

As a potential new therapeutic modality, most participants had some awareness of gene therapy, and we observed an apparent trend of increased knowledge of gene therapy with higher levels of concern.

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