Real-world treatment, dosing, and discontinuation patterns among patients treated with pegvaliase for phenylketonuria: Evidence from dispensing data.

Authors:
Lah M; Cook K; Gomes DA; Liu S; Tabatabaeepour N and 6 more

Journal:
Mol Genet Metab Rep

Publication Year: 2022

DOI:
10.1016/j.ymgmr.2022.100918

PMCID:
PMC9513695

PMID:
36176956

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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33.3% Transparent
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Evidence found in paper:

"We thank Kelly Adamski for supporting the design and development of the statistical analysis plan and coordinating the sample selection and construction process. We also thank Brian Binns and Justin Ryan from BioMarin for their assistance in supplying de-identified patient-level dispensing and case management data for this study. Funding and other disclosures statement This study was funded by BioMarin Pharmaceutical Inc. Authors EC, KL, KBW, and JVB are employees of BioMarin Pharmaceutical Inc. Authors ML and BB have received consultancy fees from BioMarin Pharmaceutical Inc. Authors NK, KC, DAG, SL, and NT are employees of Analysis Group, Inc., which has received consultancy fees from BioMarin Pharmaceutical Inc."

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Open Access
Paper is freely available to read
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Tool: rtransparent

OST Version: N/A

Last Updated: Aug 05, 2025