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

Authors:
Regier DS; Bąk A; Bausell H; O'Reilly E; Cowsert LM.

Journal:
Mol Genet Metab Rep

Publication Year: 2022

DOI:
10.1016/j.ymgmr.2022.100855

PMCID:
PMC9248220

PMID:
35782620

View on PubMed View on PMC View Publisher
Journal Information

Full Title: Mol Genet Metab Rep

Abbreviation: Mol Genet Metab Rep

Country: Unknown

Publisher: Unknown

Language: N/A

Publication Details

Subject Category: Genetics & Heredity

Available in Europe PMC: Yes

Available in PMC: Yes

PDF Available: No

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Evidence found in paper:

"Declaration of Competing Interest Heather Bausell has acted as a paid consultant for BioMarin Pharmaceutical Inc., Ultragenyx, Horizon, and Nutricia; has received speaker fees from BioMarin Pharmaceutical Inc., Ultragenyx, Vitaflo International Ltd., and Cambrooke Therapeutics; and has received financial support for travel from 10.13039/100008484BioMarin Pharmaceutical Inc and Vitaflo. Agata Bąk has received consultancy fees from BioMarin Pharmaceutical Inc. The other authors declare no competing interests."

Evidence found in paper:

"Funding BioMarin Pharmaceutical Inc. funded the development of the study design and discussion aids, conduct of the interviews, data analysis, and editorial support for the preparation of this manuscript."

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Paper is freely available to read
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Last Updated: Aug 05, 2025