Lessons learned from 5 years of pegvaliase in US clinics: A case series

Experience from the presented cases and 5 years of expert experience with pegvaliase in the real-world setting provide insight and guidance for healthcare professionals initiating and managing pegvaliase treatment in complex PKU cases. These cases demonstrate that, through comprehensive assessment and addressing barriers, pegvaliase treatment can be successful in adults with PKU, regardless of prior treatment success, age, socioeconomic, cognitive, or executive function challenges, as well as in those with comorbidities or considering pregnancy. Ongoing documentation of clinical experience is crucial for advancing the management of individuals receiving this treatment.

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Publication Details

Journal
Molecular Genetics and Metabolism Reports
Published
2024-12-26
DOI
https://doi.org/10.1016/j.ymgmr.2024.101181
Citations
1
Primary Topic
Metabolism and Genetic Disorders
Type
article
Field-Weighted Citation Impact
0.38

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article

Lessons learned from 5 years of pegvaliase in US clinics: A case series

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Lessons learned from 5 years of pegvaliase in US clinics: A case series

Gillian E. Clague, Marilyn Clifford, Laura Davis‐Keppen, Stephanie Sacharow, Erika Vucko, Melissa Lah, Bridget Wardley, Amarilis Sanchez‐Valle, Erin E. Cooney, Kirsten Havens, Leah Wessenberg, Angela Crutcher, Zineb Ammous, Tricia Bender, Hans C. Andersson
article en
1 citations

Abstract

Experience from the presented cases and 5 years of expert experience with pegvaliase in the real-world setting provide insight and guidance for healthcare professionals initiating and managing pegvaliase treatment in complex PKU cases. These cases demonstrate that, through comprehensive assessment and addressing barriers, pegvaliase treatment can be successful in adults with PKU, regardless of prior treatment success, age, socioeconomic, cognitive, or executive function challenges, as well as in those with comorbidities or considering pregnancy. Ongoing documentation of clinical experience is crucial for advancing the management of individuals receiving this treatment.

Molecular Genetics and Metabolism ReportsVol. 42
Boston Children's Hospital (US), Lurie Children's Hospital (US), University of South Dakota (US), University of South Florida (US), Tulane Medical Center (US), University Medical Center New Orleans (US), Sexual Health Clinic (FI), Doernbecher Children's Hospital (US), Cooper University Health Care (US), Shriners Hospitals for Children Medical Center - Lexington (US), Indiana University School of Medicine, The University of Texas Medical Branch at Galveston (US), Indiana University – Purdue University Indianapolis (US), BioMarin (United States) (US)
BioMarin Pharmaceutical
Openalex Percentile: Top 32%
Metabolism and Genetic Disorders
0.38
DOIOpen Access PDFOpenAlex
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